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MargaritaChic
03-21-2008, 11:42 AM
Has anyone seen any recent news regarding PTC124? I would love to see any articles you have come across.

MargaritaChic
03-21-2008, 11:42 AM
Has anyone seen any recent news regarding PTC124? I would love to see any articles you have come across.

MargaritaChic
03-21-2008, 11:42 AM
Has anyone seen any recent news regarding PTC124? I would love to see any articles you have come across.

MargaritaChic
03-21-2008, 11:42 AM
Has anyone seen any recent news regarding PTC124? I would love to see any articles you have come across.

MargaritaChic
03-21-2008, 11:42 AM
Has anyone seen any recent news regarding PTC124? I would love to see any articles you have come across.

miesl
03-21-2008, 12:53 PM
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.

I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.

Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.

-Michelle

miesl
03-21-2008, 12:53 PM
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.

I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.

Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.

-Michelle

miesl
03-21-2008, 12:53 PM
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.

I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.

Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.

-Michelle

miesl
03-21-2008, 12:53 PM
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.

I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.

Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.

-Michelle

miesl
03-21-2008, 12:53 PM
I haven't seen anything recently - but have you joined the mailing list on the PTC website? It looks like they mail out updates when they have them.
<br />
<br />I don't know much about clinical studies with infants since they are limited on quality of life feedback and spirometry - but I would mention to your CF doc that you're interested in participating in studies if your daughter qualifies. I would assume that based on the favorable Phase 2 studies, PTC will be moving to Phase 3 soon and need more people for testing.
<br />
<br />Studies are wonderful to participate in! Not only can you get experimental meds and treatment options - some of them pay quite nicely.
<br />
<br />-Michelle

NoExcuses
03-21-2008, 12:59 PM
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">


<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>

NoExcuses
03-21-2008, 12:59 PM
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">


<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>

NoExcuses
03-21-2008, 12:59 PM
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">


<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>

NoExcuses
03-21-2008, 12:59 PM
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">


<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>

NoExcuses
03-21-2008, 12:59 PM
Marla you have probably seen this before but the CFF updates this pipeline regularly <img src="i/expressions/face-icon-small-smile.gif" border="0">
<br />
<br />
<br /><a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/DrugDevelopmentPipeline/">http://www.cff.org/research/DrugDevelopmentPipeline/</a>

Faust
03-21-2008, 01:56 PM
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.

Faust
03-21-2008, 01:56 PM
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.

Faust
03-21-2008, 01:56 PM
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.

Faust
03-21-2008, 01:56 PM
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.

Faust
03-21-2008, 01:56 PM
My thoughts regarding PTC124: This will be wonderful news for the smaller percentage of CF's with mutations ending in X (from what I have been told, those are the only nonsense mutations). For the others without these mutations, it will mean nothing besides knowing others are technically being cured.
<br />
<br />
<br />

NoExcuses
03-21-2008, 02:01 PM
ya, exactly SeanDavis. notice her kid has w1282x

NoExcuses
03-21-2008, 02:01 PM
ya, exactly SeanDavis. notice her kid has w1282x

NoExcuses
03-21-2008, 02:01 PM
ya, exactly SeanDavis. notice her kid has w1282x

NoExcuses
03-21-2008, 02:01 PM
ya, exactly SeanDavis. notice her kid has w1282x

NoExcuses
03-21-2008, 02:01 PM
ya, exactly SeanDavis. notice her kid has w1282x

MargaritaChic
03-21-2008, 02:22 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>NoExcuses</b></i>

ya, exactly SeanDavis. notice her kid has w1282x</end quote></div>

Exactly, that is why I am interested. I realize it won't mean much for the majority. It could be potentially life changing for my daughter someday. <img src="i/expressions/heart.gif" border="0">

MargaritaChic
03-21-2008, 02:22 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>NoExcuses</b></i>

ya, exactly SeanDavis. notice her kid has w1282x</end quote></div>

Exactly, that is why I am interested. I realize it won't mean much for the majority. It could be potentially life changing for my daughter someday. <img src="i/expressions/heart.gif" border="0">

MargaritaChic
03-21-2008, 02:22 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>NoExcuses</b></i>

ya, exactly SeanDavis. notice her kid has w1282x</end quote></div>

Exactly, that is why I am interested. I realize it won't mean much for the majority. It could be potentially life changing for my daughter someday. <img src="i/expressions/heart.gif" border="0">

MargaritaChic
03-21-2008, 02:22 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>NoExcuses</b></i>

ya, exactly SeanDavis. notice her kid has w1282x</end quote>

Exactly, that is why I am interested. I realize it won't mean much for the majority. It could be potentially life changing for my daughter someday. <img src="i/expressions/heart.gif" border="0">

MargaritaChic
03-21-2008, 02:22 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>NoExcuses</b></i>
<br />
<br />ya, exactly SeanDavis. notice her kid has w1282x</end quote>
<br />
<br />Exactly, that is why I am interested. I realize it won't mean much for the majority. It could be potentially life changing for my daughter someday. <img src="i/expressions/heart.gif" border="0">
<br />
<br />

NoExcuses
03-21-2008, 02:29 PM
a win for any CFer is a win in my mind. <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0">

NoExcuses
03-21-2008, 02:29 PM
a win for any CFer is a win in my mind. <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0">

NoExcuses
03-21-2008, 02:29 PM
a win for any CFer is a win in my mind. <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0">

NoExcuses
03-21-2008, 02:29 PM
a win for any CFer is a win in my mind. <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0">

NoExcuses
03-21-2008, 02:29 PM
a win for any CFer is a win in my mind. <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0"> <img src="i/expressions/face-icon-small-smile.gif" border="0">

Faust
03-22-2008, 04:41 AM
I agree. It will never be a negative thing when ANY CF if cured. Especially the younger ones. I just wish it was more inclusive is all. But that isn't how life works.


As long as I can keep drinking my Sam Adams and have fun while I slowly die, that's ok too.

Faust
03-22-2008, 04:41 AM
I agree. It will never be a negative thing when ANY CF if cured. Especially the younger ones. I just wish it was more inclusive is all. But that isn't how life works.


As long as I can keep drinking my Sam Adams and have fun while I slowly die, that's ok too.

Faust
03-22-2008, 04:41 AM
I agree. It will never be a negative thing when ANY CF if cured. Especially the younger ones. I just wish it was more inclusive is all. But that isn't how life works.


As long as I can keep drinking my Sam Adams and have fun while I slowly die, that's ok too.

Faust
03-22-2008, 04:41 AM
I agree. It will never be a negative thing when ANY CF if cured. Especially the younger ones. I just wish it was more inclusive is all. But that isn't how life works.


As long as I can keep drinking my Sam Adams and have fun while I slowly die, that's ok too.

Faust
03-22-2008, 04:41 AM
I agree. It will never be a negative thing when ANY CF if cured. Especially the younger ones. I just wish it was more inclusive is all. But that isn't how life works.
<br />
<br />
<br />As long as I can keep drinking my Sam Adams and have fun while I slowly die, that's ok too.
<br />
<br />

Jane
03-22-2008, 06:03 PM
Maria, thanks for posting this. I guess I haven't paid enough attention. I realize now my boys would benefit as well.

As Amy said "a win...".

Jane
03-22-2008, 06:03 PM
Maria, thanks for posting this. I guess I haven't paid enough attention. I realize now my boys would benefit as well.

As Amy said "a win...".

Jane
03-22-2008, 06:03 PM
Maria, thanks for posting this. I guess I haven't paid enough attention. I realize now my boys would benefit as well.

As Amy said "a win...".

Jane
03-22-2008, 06:03 PM
Maria, thanks for posting this. I guess I haven't paid enough attention. I realize now my boys would benefit as well.

As Amy said "a win...".

Jane
03-22-2008, 06:03 PM
Maria, thanks for posting this. I guess I haven't paid enough attention. I realize now my boys would benefit as well.
<br />
<br />As Amy said "a win...".

janddburke
04-15-2008, 08:09 PM
considering it's potential, news of this drug has been rather hard to find.
what do you think that means?

janddburke
04-15-2008, 08:09 PM
considering it's potential, news of this drug has been rather hard to find.
what do you think that means?

janddburke
04-15-2008, 08:09 PM
considering it's potential, news of this drug has been rather hard to find.
what do you think that means?

janddburke
04-15-2008, 08:09 PM
considering it's potential, news of this drug has been rather hard to find.
what do you think that means?

janddburke
04-15-2008, 08:09 PM
considering it's potential, news of this drug has been rather hard to find.
<br />what do you think that means?

Jane
04-15-2008, 09:18 PM
We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.

QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm

Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?

Jane
04-15-2008, 09:18 PM
We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.

QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm

Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?

Jane
04-15-2008, 09:18 PM
We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.

QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm

Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?

Jane
04-15-2008, 09:18 PM
We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.

QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm

Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?

Jane
04-15-2008, 09:18 PM
We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.
<br />
<br />QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm
<br />
<br />Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?

beccasmom
04-15-2008, 10:06 PM
The Cystic fibrosis foundation had a press release a few weeks ago about this
www.cff.org/aboutCFFoundation/NewsEvents/index.cfm?ID=8476&Type=1670

There are some links to other information

beccasmom
04-15-2008, 10:06 PM
The Cystic fibrosis foundation had a press release a few weeks ago about this
www.cff.org/aboutCFFoundation/NewsEvents/index.cfm?ID=8476&Type=1670

There are some links to other information

beccasmom
04-15-2008, 10:06 PM
The Cystic fibrosis foundation had a press release a few weeks ago about this
www.cff.org/aboutCFFoundation/NewsEvents/index.cfm?ID=8476&Type=1670

There are some links to other information

beccasmom
04-15-2008, 10:06 PM
The Cystic fibrosis foundation had a press release a few weeks ago about this
www.cff.org/aboutCFFoundation/NewsEvents/index.cfm?ID=8476&Type=1670

There are some links to other information

beccasmom
04-15-2008, 10:06 PM
The Cystic fibrosis foundation had a press release a few weeks ago about this
<br />www.cff.org/aboutCFFoundation/NewsEvents/index.cfm?ID=8476&Type=1670
<br />
<br />There are some links to other information

hopefullmom
06-09-2008, 01:56 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:56 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:56 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:56 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:56 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
<br />mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:57 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:57 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:57 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:57 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
mom to Maci W1282X and DF508

hopefullmom
06-09-2008, 01:57 PM
I am writing to you all to tell you I got a call from the national foundation last week on ptc124 and they have used all there funding for the drug, and need people raising money so this drug can stay in trial. I was devastated. I know that if we raise money we can fund this drug directly. I have a child that this could possibly help.
<br />mom to Maci W1282X and DF508

Diane
06-09-2008, 04:47 PM
Thats not exactly the news i wanted to hear. If you have any ideas on how to raise the money please let me know, i'll do what i can.

Diane
06-09-2008, 04:47 PM
Thats not exactly the news i wanted to hear. If you have any ideas on how to raise the money please let me know, i'll do what i can.

Diane
06-09-2008, 04:47 PM
Thats not exactly the news i wanted to hear. If you have any ideas on how to raise the money please let me know, i'll do what i can.

Diane
06-09-2008, 04:47 PM
Thats not exactly the news i wanted to hear. If you have any ideas on how to raise the money please let me know, i'll do what i can.

Diane
06-09-2008, 04:47 PM
Thats not exactly the news i wanted to hear. If you have any ideas on how to raise the money please let me know, i'll do what i can.

princessjdc
06-09-2008, 05:01 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Jane</b></i>

We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.



QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm



Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?</end quote></div>

When I asked my doc about PTC 124 he didnt sound too enthused about the drug. Heres my thought when it comes out I will diffantly make sure Im on it whether or not my doc thinks much of it or not. But I hope that they are able to continue to fund PTC 124 cause I think I will benefit from it greatly both my mutations is G542X. <img src="i/expressions/face-icon-small-smile.gif" border="0">

princessjdc
06-09-2008, 05:01 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Jane</b></i>

We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.



QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm



Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?</end quote></div>

When I asked my doc about PTC 124 he didnt sound too enthused about the drug. Heres my thought when it comes out I will diffantly make sure Im on it whether or not my doc thinks much of it or not. But I hope that they are able to continue to fund PTC 124 cause I think I will benefit from it greatly both my mutations is G542X. <img src="i/expressions/face-icon-small-smile.gif" border="0">

princessjdc
06-09-2008, 05:01 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Jane</b></i>

We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.



QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm



Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?</end quote></div>

When I asked my doc about PTC 124 he didnt sound too enthused about the drug. Heres my thought when it comes out I will diffantly make sure Im on it whether or not my doc thinks much of it or not. But I hope that they are able to continue to fund PTC 124 cause I think I will benefit from it greatly both my mutations is G542X. <img src="i/expressions/face-icon-small-smile.gif" border="0">

princessjdc
06-09-2008, 05:01 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Jane</b></i>

We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.



QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm



Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?</end quote>

When I asked my doc about PTC 124 he didnt sound too enthused about the drug. Heres my thought when it comes out I will diffantly make sure Im on it whether or not my doc thinks much of it or not. But I hope that they are able to continue to fund PTC 124 cause I think I will benefit from it greatly both my mutations is G542X. <img src="i/expressions/face-icon-small-smile.gif" border="0">

princessjdc
06-09-2008, 05:01 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>Jane</b></i>
<br />
<br />We asked our doctor about it when we were in clinic. She was very vague and then told us to be careful of things you read on the internet.
<br />
<br />
<br />
<br />QUOTE "you wouldn't believe some of the things that my patients find in CF chat rooms. She said those people are very bitter. You can read all kinds of crazy stuff". hmmmm
<br />
<br />
<br />
<br />Honestly- if I didn't come here, I wouldn't know much. Are the docs just being cautious?</end quote>
<br />
<br />When I asked my doc about PTC 124 he didnt sound too enthused about the drug. Heres my thought when it comes out I will diffantly make sure Im on it whether or not my doc thinks much of it or not. But I hope that they are able to continue to fund PTC 124 cause I think I will benefit from it greatly both my mutations is G542X. <img src="i/expressions/face-icon-small-smile.gif" border="0">
<br />

lightNlife
06-09-2008, 06:23 PM
I mentioned PTC 124 in the article I wrote for this month's cysticfibrosis.com newsletter.

lightNlife
06-09-2008, 06:23 PM
I mentioned PTC 124 in the article I wrote for this month's cysticfibrosis.com newsletter.

lightNlife
06-09-2008, 06:23 PM
I mentioned PTC 124 in the article I wrote for this month's cysticfibrosis.com newsletter.

lightNlife
06-09-2008, 06:23 PM
I mentioned PTC 124 in the article I wrote for this month's cysticfibrosis.com newsletter.

lightNlife
06-09-2008, 06:23 PM
I mentioned PTC 124 in the article I wrote for this month's cysticfibrosis.com newsletter.

cfsucks
06-09-2008, 08:06 PM
my doctor hadn't even heard of it

cfsucks
06-09-2008, 08:06 PM
my doctor hadn't even heard of it

cfsucks
06-09-2008, 08:06 PM
my doctor hadn't even heard of it

cfsucks
06-09-2008, 08:06 PM
my doctor hadn't even heard of it

cfsucks
06-09-2008, 08:06 PM
my doctor hadn't even heard of it

hopefullmom
06-10-2008, 05:14 AM
I am going to try and get a website about this going, I am also going to use the money that I raise for the walk toward this drug, which last year was around 15,000, which only puts a small dent in the 10 million they need. I am also writing letters to every TV celebrity I can think of, I just pray that someone will be generous enough to at least consider helping. This is why it is so important for people to be involved in the walk and raising awareness. Every little bit counts. I will do everything I can to try and help this drug go through, they have had great results with it. Thanks, Mom to Maci 5yrs old

hopefullmom
06-10-2008, 05:14 AM
I am going to try and get a website about this going, I am also going to use the money that I raise for the walk toward this drug, which last year was around 15,000, which only puts a small dent in the 10 million they need. I am also writing letters to every TV celebrity I can think of, I just pray that someone will be generous enough to at least consider helping. This is why it is so important for people to be involved in the walk and raising awareness. Every little bit counts. I will do everything I can to try and help this drug go through, they have had great results with it. Thanks, Mom to Maci 5yrs old

hopefullmom
06-10-2008, 05:14 AM
I am going to try and get a website about this going, I am also going to use the money that I raise for the walk toward this drug, which last year was around 15,000, which only puts a small dent in the 10 million they need. I am also writing letters to every TV celebrity I can think of, I just pray that someone will be generous enough to at least consider helping. This is why it is so important for people to be involved in the walk and raising awareness. Every little bit counts. I will do everything I can to try and help this drug go through, they have had great results with it. Thanks, Mom to Maci 5yrs old

hopefullmom
06-10-2008, 05:14 AM
I am going to try and get a website about this going, I am also going to use the money that I raise for the walk toward this drug, which last year was around 15,000, which only puts a small dent in the 10 million they need. I am also writing letters to every TV celebrity I can think of, I just pray that someone will be generous enough to at least consider helping. This is why it is so important for people to be involved in the walk and raising awareness. Every little bit counts. I will do everything I can to try and help this drug go through, they have had great results with it. Thanks, Mom to Maci 5yrs old

hopefullmom
06-10-2008, 05:14 AM
I am going to try and get a website about this going, I am also going to use the money that I raise for the walk toward this drug, which last year was around 15,000, which only puts a small dent in the 10 million they need. I am also writing letters to every TV celebrity I can think of, I just pray that someone will be generous enough to at least consider helping. This is why it is so important for people to be involved in the walk and raising awareness. Every little bit counts. I will do everything I can to try and help this drug go through, they have had great results with it. Thanks, Mom to Maci 5yrs old

Juliet
06-13-2008, 02:51 PM
This was a press release posted on 6-12-08.

<a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com/4.1_news.aspx#PR06122008
">http://www.ptcbio.com/4.1_news.aspx#PR06122008
</a>
PTC THERAPEUTICS ANNOUNCES DATA FROM ADDITIONAL CLINICAL STUDIES OF PTC124 IN CYSTIC FIBROSIS CONFIRMING ACTIVITY
- Data Presented at the 31st European Cystic Fibrosis Conference -

South Plainfield, NJ and Prague, Czech Republic- June 12, 2008 - PTC Therapeutics, Inc. (PTC) today announced promising new data from two studies of PTC124 in cystic fibrosis (CF). Results from an Israeli Phase 2a extension study evaluating three months of oral PTC124 treatment in adult patients with nonsense-mutation-mediated CF demonstrated statistically significant improvements in the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and a statistically significant mean [28%] decrease in the frequency of cough, one of the most prominent and burdensome CF-related symptoms. Results from the Hadassah University Hospital in Israel were presented today by Michael Wilschanski, M.D., Director, Pediatric Gastroenterology, and by Eitan Kerem, M.D., principal investigator and head of the Department of Pediatrics and Cystic Fibrosis Center. Separately, results from a European study evaluating 14-day courses of PTC124 in pediatric patients with nonsense-mutation-mediated CF confirmed the CFTR activity observed in previous short-term studies in adult patients. Data from the European study were presented today by Isabelle Sermet-Gaudelus, M.D., Ph.D., principal investigator at l'H˘pital Necker-Enfants Malade.

Patients with CF lack adequate levels of the CFTR protein, a chloride channel that maintains proper hydration of epithelial surfaces in the lung, pancreas, and liver. Patients with nonsense-mutation-mediated CF generally have a severe form of CF because virtually no CFTR protein is produced. Previous studies of PTC124 adult patients with CF evaluated nasal transepithelial potential difference (TEPD) as a surrogate for the presence and activity of the CFTR protein. Across the short- and long-term clinical trials at high and low doses of PTC124, TEPD assessments showed statistically significant improvements of mean CFTR-dependent chloride secretion in the airways.

The Phase 2a extension study in Israel assessed 3 months of oral PTC124 therapy at two different dose levels in 19 adult men and women with nonsense-mutation-mediated CF who had participated in a prior short-term PTC124 Phase 2a study. More than 90% of the patients had chronic CF-related lung infection and also had CF-induced pancreatic insufficiency. Results from the study showed that treatment with PTC124 resulted in statistically significant (p<0.001) improvements in CFTR function as measured by TEPD in both dose groups. The proportion of patients showing improvement in TEPD chloride secretion increased over time in the extension study. Trends towards improvements in mean FEV1 and FVC values were observed. Baseline data showed that CF patients cough a remarkable 643 times per day on average, with a range of 324 to 1,569 coughs per day. In comparison, healthy individuals generally cough fewer than 16 times per day, according to the European Respiratory Journal (Hsu 1994). Patients receiving PTC124 experienced a mean [28%] decrease in cough frequency by the end of three months of therapy (p<0.01). PTC124 was generally well tolerated, resulting in excellent mean compliance with the treatment regimen (>90%).

"Three months of treatment with PTC124 in nonsense-mutation-mediated CF patients was associated with time-dependent improvements in nasal TEPD chloride conductance, pulmonary function and cough - important markers suggesting the potential for benefit in patients with CF," stated Dr. Wilschanski. "PTC124 increases CFTR-mediated chloride secretion in patients with a variety of nonsense mutation types, which suggests a broad spectrum of activity across one of the major subpopulations in CF."

Dr. Kerem added, "The impact on cough that we observed in this study by objective measurement is particularly notable given that cough is one of the major symptomatic manifestations of the underlying disease process in CF. The reduction in cough achieved with PTC124 in this three-month study suggests secondary clinical effects of drug activity. Based on these findings, we believe that objective measurement of cough may offer a meaningful new way to evaluate drug efficacy in future CF clinical trials."

In a separate Phase 2a study in Europe, data are currently available from 21 children who received two 14-day treatment courses of oral PTC124 therapy at two different dose levels. Patients ranged in age from six to 18 years. All had nonsense-mutation-mediated disease, pathological lung infection, and pancreatic insufficiency. Statistically significant (p<0.05) increases in the proportion of epithelial cells showing surface staining with the CFTR protein were observed. In addition, statistically significant (p<0.05) improvements in CFTR-mediated chloride conductance as measured by TEPD were evident. PTC124 was generally well tolerated in pediatric patients and mean compliance with treatment was excellent (>95%).

"We are encouraged to see the activity of PTC124 observed in adults reproduced in a pediatric population in France and Belgium," commented Dr. Sermet-Gaudelus. "PTC124 causes the missing protein to be made, to be located in the right place in the cell, and to have functional effect. Combined with the generally well-tolerated profile of PTC124, we believe these data support inclusion of pediatric patients in future clinical trials."

"We are gratified by these results from our Israeli, French, and Belgian investigators, which demonstrated the activity of PTC124 in patients across a variety of age ranges, geographies, and nonsense mutation types," stated Langdon Miller, M.D., Chief Medical Officer of PTC. "These data offer the basis for initiating a randomized, controlled Phase 2b study later this year to evaluate the clinical benefit of PTC124 in adults and children with nonsense-mutation-mediated CF."

ABOUT CYSTIC FIBROSIS
Cystic fibrosis (CF) is among the most common life-threatening genetic disorders worldwide. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and, according to the European Cystic Fibrosis Foundation, it affects a similar number of patients in Europe. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10% of patients in the United States. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments that address the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (www.cff.org).

ABOUT PTC124
PTC124 is an orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce functional protein across a variety of nonsense mutation types. Across all clinical studies to date, PTC124 has been generally well tolerated and has achieved target plasma concentrations that have been associated with activity in preclinical models. PTC124 is currently in Phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mediated genetic disorders may provide clinical benefits.

ABOUT PTC THERAPEUTICS INC.
PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-molecules (GEMS) technology platform, which has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Pfizer, Celgene, CV Therapeutics and Schering-Plough. For more information, visit the company's website, www.ptcbio.com.

Juliet
06-13-2008, 02:51 PM
This was a press release posted on 6-12-08.

<a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com/4.1_news.aspx#PR06122008
">http://www.ptcbio.com/4.1_news.aspx#PR06122008
</a>
PTC THERAPEUTICS ANNOUNCES DATA FROM ADDITIONAL CLINICAL STUDIES OF PTC124 IN CYSTIC FIBROSIS CONFIRMING ACTIVITY
- Data Presented at the 31st European Cystic Fibrosis Conference -

South Plainfield, NJ and Prague, Czech Republic- June 12, 2008 - PTC Therapeutics, Inc. (PTC) today announced promising new data from two studies of PTC124 in cystic fibrosis (CF). Results from an Israeli Phase 2a extension study evaluating three months of oral PTC124 treatment in adult patients with nonsense-mutation-mediated CF demonstrated statistically significant improvements in the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and a statistically significant mean [28%] decrease in the frequency of cough, one of the most prominent and burdensome CF-related symptoms. Results from the Hadassah University Hospital in Israel were presented today by Michael Wilschanski, M.D., Director, Pediatric Gastroenterology, and by Eitan Kerem, M.D., principal investigator and head of the Department of Pediatrics and Cystic Fibrosis Center. Separately, results from a European study evaluating 14-day courses of PTC124 in pediatric patients with nonsense-mutation-mediated CF confirmed the CFTR activity observed in previous short-term studies in adult patients. Data from the European study were presented today by Isabelle Sermet-Gaudelus, M.D., Ph.D., principal investigator at l'H˘pital Necker-Enfants Malade.

Patients with CF lack adequate levels of the CFTR protein, a chloride channel that maintains proper hydration of epithelial surfaces in the lung, pancreas, and liver. Patients with nonsense-mutation-mediated CF generally have a severe form of CF because virtually no CFTR protein is produced. Previous studies of PTC124 adult patients with CF evaluated nasal transepithelial potential difference (TEPD) as a surrogate for the presence and activity of the CFTR protein. Across the short- and long-term clinical trials at high and low doses of PTC124, TEPD assessments showed statistically significant improvements of mean CFTR-dependent chloride secretion in the airways.

The Phase 2a extension study in Israel assessed 3 months of oral PTC124 therapy at two different dose levels in 19 adult men and women with nonsense-mutation-mediated CF who had participated in a prior short-term PTC124 Phase 2a study. More than 90% of the patients had chronic CF-related lung infection and also had CF-induced pancreatic insufficiency. Results from the study showed that treatment with PTC124 resulted in statistically significant (p<0.001) improvements in CFTR function as measured by TEPD in both dose groups. The proportion of patients showing improvement in TEPD chloride secretion increased over time in the extension study. Trends towards improvements in mean FEV1 and FVC values were observed. Baseline data showed that CF patients cough a remarkable 643 times per day on average, with a range of 324 to 1,569 coughs per day. In comparison, healthy individuals generally cough fewer than 16 times per day, according to the European Respiratory Journal (Hsu 1994). Patients receiving PTC124 experienced a mean [28%] decrease in cough frequency by the end of three months of therapy (p<0.01). PTC124 was generally well tolerated, resulting in excellent mean compliance with the treatment regimen (>90%).

"Three months of treatment with PTC124 in nonsense-mutation-mediated CF patients was associated with time-dependent improvements in nasal TEPD chloride conductance, pulmonary function and cough - important markers suggesting the potential for benefit in patients with CF," stated Dr. Wilschanski. "PTC124 increases CFTR-mediated chloride secretion in patients with a variety of nonsense mutation types, which suggests a broad spectrum of activity across one of the major subpopulations in CF."

Dr. Kerem added, "The impact on cough that we observed in this study by objective measurement is particularly notable given that cough is one of the major symptomatic manifestations of the underlying disease process in CF. The reduction in cough achieved with PTC124 in this three-month study suggests secondary clinical effects of drug activity. Based on these findings, we believe that objective measurement of cough may offer a meaningful new way to evaluate drug efficacy in future CF clinical trials."

In a separate Phase 2a study in Europe, data are currently available from 21 children who received two 14-day treatment courses of oral PTC124 therapy at two different dose levels. Patients ranged in age from six to 18 years. All had nonsense-mutation-mediated disease, pathological lung infection, and pancreatic insufficiency. Statistically significant (p<0.05) increases in the proportion of epithelial cells showing surface staining with the CFTR protein were observed. In addition, statistically significant (p<0.05) improvements in CFTR-mediated chloride conductance as measured by TEPD were evident. PTC124 was generally well tolerated in pediatric patients and mean compliance with treatment was excellent (>95%).

"We are encouraged to see the activity of PTC124 observed in adults reproduced in a pediatric population in France and Belgium," commented Dr. Sermet-Gaudelus. "PTC124 causes the missing protein to be made, to be located in the right place in the cell, and to have functional effect. Combined with the generally well-tolerated profile of PTC124, we believe these data support inclusion of pediatric patients in future clinical trials."

"We are gratified by these results from our Israeli, French, and Belgian investigators, which demonstrated the activity of PTC124 in patients across a variety of age ranges, geographies, and nonsense mutation types," stated Langdon Miller, M.D., Chief Medical Officer of PTC. "These data offer the basis for initiating a randomized, controlled Phase 2b study later this year to evaluate the clinical benefit of PTC124 in adults and children with nonsense-mutation-mediated CF."

ABOUT CYSTIC FIBROSIS
Cystic fibrosis (CF) is among the most common life-threatening genetic disorders worldwide. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and, according to the European Cystic Fibrosis Foundation, it affects a similar number of patients in Europe. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10% of patients in the United States. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments that address the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (www.cff.org).

ABOUT PTC124
PTC124 is an orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce functional protein across a variety of nonsense mutation types. Across all clinical studies to date, PTC124 has been generally well tolerated and has achieved target plasma concentrations that have been associated with activity in preclinical models. PTC124 is currently in Phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mediated genetic disorders may provide clinical benefits.

ABOUT PTC THERAPEUTICS INC.
PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-molecules (GEMS) technology platform, which has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Pfizer, Celgene, CV Therapeutics and Schering-Plough. For more information, visit the company's website, www.ptcbio.com.

Juliet
06-13-2008, 02:51 PM
This was a press release posted on 6-12-08.

<a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com/4.1_news.aspx#PR06122008
">http://www.ptcbio.com/4.1_news.aspx#PR06122008
</a>
PTC THERAPEUTICS ANNOUNCES DATA FROM ADDITIONAL CLINICAL STUDIES OF PTC124 IN CYSTIC FIBROSIS CONFIRMING ACTIVITY
- Data Presented at the 31st European Cystic Fibrosis Conference -

South Plainfield, NJ and Prague, Czech Republic- June 12, 2008 - PTC Therapeutics, Inc. (PTC) today announced promising new data from two studies of PTC124 in cystic fibrosis (CF). Results from an Israeli Phase 2a extension study evaluating three months of oral PTC124 treatment in adult patients with nonsense-mutation-mediated CF demonstrated statistically significant improvements in the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and a statistically significant mean [28%] decrease in the frequency of cough, one of the most prominent and burdensome CF-related symptoms. Results from the Hadassah University Hospital in Israel were presented today by Michael Wilschanski, M.D., Director, Pediatric Gastroenterology, and by Eitan Kerem, M.D., principal investigator and head of the Department of Pediatrics and Cystic Fibrosis Center. Separately, results from a European study evaluating 14-day courses of PTC124 in pediatric patients with nonsense-mutation-mediated CF confirmed the CFTR activity observed in previous short-term studies in adult patients. Data from the European study were presented today by Isabelle Sermet-Gaudelus, M.D., Ph.D., principal investigator at l'H˘pital Necker-Enfants Malade.

Patients with CF lack adequate levels of the CFTR protein, a chloride channel that maintains proper hydration of epithelial surfaces in the lung, pancreas, and liver. Patients with nonsense-mutation-mediated CF generally have a severe form of CF because virtually no CFTR protein is produced. Previous studies of PTC124 adult patients with CF evaluated nasal transepithelial potential difference (TEPD) as a surrogate for the presence and activity of the CFTR protein. Across the short- and long-term clinical trials at high and low doses of PTC124, TEPD assessments showed statistically significant improvements of mean CFTR-dependent chloride secretion in the airways.

The Phase 2a extension study in Israel assessed 3 months of oral PTC124 therapy at two different dose levels in 19 adult men and women with nonsense-mutation-mediated CF who had participated in a prior short-term PTC124 Phase 2a study. More than 90% of the patients had chronic CF-related lung infection and also had CF-induced pancreatic insufficiency. Results from the study showed that treatment with PTC124 resulted in statistically significant (p<0.001) improvements in CFTR function as measured by TEPD in both dose groups. The proportion of patients showing improvement in TEPD chloride secretion increased over time in the extension study. Trends towards improvements in mean FEV1 and FVC values were observed. Baseline data showed that CF patients cough a remarkable 643 times per day on average, with a range of 324 to 1,569 coughs per day. In comparison, healthy individuals generally cough fewer than 16 times per day, according to the European Respiratory Journal (Hsu 1994). Patients receiving PTC124 experienced a mean [28%] decrease in cough frequency by the end of three months of therapy (p<0.01). PTC124 was generally well tolerated, resulting in excellent mean compliance with the treatment regimen (>90%).

"Three months of treatment with PTC124 in nonsense-mutation-mediated CF patients was associated with time-dependent improvements in nasal TEPD chloride conductance, pulmonary function and cough - important markers suggesting the potential for benefit in patients with CF," stated Dr. Wilschanski. "PTC124 increases CFTR-mediated chloride secretion in patients with a variety of nonsense mutation types, which suggests a broad spectrum of activity across one of the major subpopulations in CF."

Dr. Kerem added, "The impact on cough that we observed in this study by objective measurement is particularly notable given that cough is one of the major symptomatic manifestations of the underlying disease process in CF. The reduction in cough achieved with PTC124 in this three-month study suggests secondary clinical effects of drug activity. Based on these findings, we believe that objective measurement of cough may offer a meaningful new way to evaluate drug efficacy in future CF clinical trials."

In a separate Phase 2a study in Europe, data are currently available from 21 children who received two 14-day treatment courses of oral PTC124 therapy at two different dose levels. Patients ranged in age from six to 18 years. All had nonsense-mutation-mediated disease, pathological lung infection, and pancreatic insufficiency. Statistically significant (p<0.05) increases in the proportion of epithelial cells showing surface staining with the CFTR protein were observed. In addition, statistically significant (p<0.05) improvements in CFTR-mediated chloride conductance as measured by TEPD were evident. PTC124 was generally well tolerated in pediatric patients and mean compliance with treatment was excellent (>95%).

"We are encouraged to see the activity of PTC124 observed in adults reproduced in a pediatric population in France and Belgium," commented Dr. Sermet-Gaudelus. "PTC124 causes the missing protein to be made, to be located in the right place in the cell, and to have functional effect. Combined with the generally well-tolerated profile of PTC124, we believe these data support inclusion of pediatric patients in future clinical trials."

"We are gratified by these results from our Israeli, French, and Belgian investigators, which demonstrated the activity of PTC124 in patients across a variety of age ranges, geographies, and nonsense mutation types," stated Langdon Miller, M.D., Chief Medical Officer of PTC. "These data offer the basis for initiating a randomized, controlled Phase 2b study later this year to evaluate the clinical benefit of PTC124 in adults and children with nonsense-mutation-mediated CF."

ABOUT CYSTIC FIBROSIS
Cystic fibrosis (CF) is among the most common life-threatening genetic disorders worldwide. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and, according to the European Cystic Fibrosis Foundation, it affects a similar number of patients in Europe. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10% of patients in the United States. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments that address the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (www.cff.org).

ABOUT PTC124
PTC124 is an orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce functional protein across a variety of nonsense mutation types. Across all clinical studies to date, PTC124 has been generally well tolerated and has achieved target plasma concentrations that have been associated with activity in preclinical models. PTC124 is currently in Phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mediated genetic disorders may provide clinical benefits.

ABOUT PTC THERAPEUTICS INC.
PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-molecules (GEMS) technology platform, which has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Pfizer, Celgene, CV Therapeutics and Schering-Plough. For more information, visit the company's website, www.ptcbio.com.

Juliet
06-13-2008, 02:51 PM
This was a press release posted on 6-12-08.

<a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com/4.1_news.aspx#PR06122008
">http://www.ptcbio.com/4.1_news.aspx#PR06122008
</a>
PTC THERAPEUTICS ANNOUNCES DATA FROM ADDITIONAL CLINICAL STUDIES OF PTC124 IN CYSTIC FIBROSIS CONFIRMING ACTIVITY
- Data Presented at the 31st European Cystic Fibrosis Conference -

South Plainfield, NJ and Prague, Czech Republic- June 12, 2008 - PTC Therapeutics, Inc. (PTC) today announced promising new data from two studies of PTC124 in cystic fibrosis (CF). Results from an Israeli Phase 2a extension study evaluating three months of oral PTC124 treatment in adult patients with nonsense-mutation-mediated CF demonstrated statistically significant improvements in the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and a statistically significant mean [28%] decrease in the frequency of cough, one of the most prominent and burdensome CF-related symptoms. Results from the Hadassah University Hospital in Israel were presented today by Michael Wilschanski, M.D., Director, Pediatric Gastroenterology, and by Eitan Kerem, M.D., principal investigator and head of the Department of Pediatrics and Cystic Fibrosis Center. Separately, results from a European study evaluating 14-day courses of PTC124 in pediatric patients with nonsense-mutation-mediated CF confirmed the CFTR activity observed in previous short-term studies in adult patients. Data from the European study were presented today by Isabelle Sermet-Gaudelus, M.D., Ph.D., principal investigator at l'H˘pital Necker-Enfants Malade.

Patients with CF lack adequate levels of the CFTR protein, a chloride channel that maintains proper hydration of epithelial surfaces in the lung, pancreas, and liver. Patients with nonsense-mutation-mediated CF generally have a severe form of CF because virtually no CFTR protein is produced. Previous studies of PTC124 adult patients with CF evaluated nasal transepithelial potential difference (TEPD) as a surrogate for the presence and activity of the CFTR protein. Across the short- and long-term clinical trials at high and low doses of PTC124, TEPD assessments showed statistically significant improvements of mean CFTR-dependent chloride secretion in the airways.

The Phase 2a extension study in Israel assessed 3 months of oral PTC124 therapy at two different dose levels in 19 adult men and women with nonsense-mutation-mediated CF who had participated in a prior short-term PTC124 Phase 2a study. More than 90% of the patients had chronic CF-related lung infection and also had CF-induced pancreatic insufficiency. Results from the study showed that treatment with PTC124 resulted in statistically significant (p<0.001) improvements in CFTR function as measured by TEPD in both dose groups. The proportion of patients showing improvement in TEPD chloride secretion increased over time in the extension study. Trends towards improvements in mean FEV1 and FVC values were observed. Baseline data showed that CF patients cough a remarkable 643 times per day on average, with a range of 324 to 1,569 coughs per day. In comparison, healthy individuals generally cough fewer than 16 times per day, according to the European Respiratory Journal (Hsu 1994). Patients receiving PTC124 experienced a mean [28%] decrease in cough frequency by the end of three months of therapy (p<0.01). PTC124 was generally well tolerated, resulting in excellent mean compliance with the treatment regimen (>90%).

"Three months of treatment with PTC124 in nonsense-mutation-mediated CF patients was associated with time-dependent improvements in nasal TEPD chloride conductance, pulmonary function and cough - important markers suggesting the potential for benefit in patients with CF," stated Dr. Wilschanski. "PTC124 increases CFTR-mediated chloride secretion in patients with a variety of nonsense mutation types, which suggests a broad spectrum of activity across one of the major subpopulations in CF."

Dr. Kerem added, "The impact on cough that we observed in this study by objective measurement is particularly notable given that cough is one of the major symptomatic manifestations of the underlying disease process in CF. The reduction in cough achieved with PTC124 in this three-month study suggests secondary clinical effects of drug activity. Based on these findings, we believe that objective measurement of cough may offer a meaningful new way to evaluate drug efficacy in future CF clinical trials."

In a separate Phase 2a study in Europe, data are currently available from 21 children who received two 14-day treatment courses of oral PTC124 therapy at two different dose levels. Patients ranged in age from six to 18 years. All had nonsense-mutation-mediated disease, pathological lung infection, and pancreatic insufficiency. Statistically significant (p<0.05) increases in the proportion of epithelial cells showing surface staining with the CFTR protein were observed. In addition, statistically significant (p<0.05) improvements in CFTR-mediated chloride conductance as measured by TEPD were evident. PTC124 was generally well tolerated in pediatric patients and mean compliance with treatment was excellent (>95%).

"We are encouraged to see the activity of PTC124 observed in adults reproduced in a pediatric population in France and Belgium," commented Dr. Sermet-Gaudelus. "PTC124 causes the missing protein to be made, to be located in the right place in the cell, and to have functional effect. Combined with the generally well-tolerated profile of PTC124, we believe these data support inclusion of pediatric patients in future clinical trials."

"We are gratified by these results from our Israeli, French, and Belgian investigators, which demonstrated the activity of PTC124 in patients across a variety of age ranges, geographies, and nonsense mutation types," stated Langdon Miller, M.D., Chief Medical Officer of PTC. "These data offer the basis for initiating a randomized, controlled Phase 2b study later this year to evaluate the clinical benefit of PTC124 in adults and children with nonsense-mutation-mediated CF."

ABOUT CYSTIC FIBROSIS
Cystic fibrosis (CF) is among the most common life-threatening genetic disorders worldwide. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and, according to the European Cystic Fibrosis Foundation, it affects a similar number of patients in Europe. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10% of patients in the United States. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments that address the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (www.cff.org).

ABOUT PTC124
PTC124 is an orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce functional protein across a variety of nonsense mutation types. Across all clinical studies to date, PTC124 has been generally well tolerated and has achieved target plasma concentrations that have been associated with activity in preclinical models. PTC124 is currently in Phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mediated genetic disorders may provide clinical benefits.

ABOUT PTC THERAPEUTICS INC.
PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-molecules (GEMS) technology platform, which has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Pfizer, Celgene, CV Therapeutics and Schering-Plough. For more information, visit the company's website, www.ptcbio.com.

Juliet
06-13-2008, 02:51 PM
This was a press release posted on 6-12-08.
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<br /><a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com/4.1_news.aspx#PR06122008
">http://www.ptcbio.com/4.1_news.aspx#PR06122008
</a><br />
<br />PTC THERAPEUTICS ANNOUNCES DATA FROM ADDITIONAL CLINICAL STUDIES OF PTC124 IN CYSTIC FIBROSIS CONFIRMING ACTIVITY
<br />- Data Presented at the 31st European Cystic Fibrosis Conference -
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<br />South Plainfield, NJ and Prague, Czech Republic- June 12, 2008 - PTC Therapeutics, Inc. (PTC) today announced promising new data from two studies of PTC124 in cystic fibrosis (CF). Results from an Israeli Phase 2a extension study evaluating three months of oral PTC124 treatment in adult patients with nonsense-mutation-mediated CF demonstrated statistically significant improvements in the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and a statistically significant mean [28%] decrease in the frequency of cough, one of the most prominent and burdensome CF-related symptoms. Results from the Hadassah University Hospital in Israel were presented today by Michael Wilschanski, M.D., Director, Pediatric Gastroenterology, and by Eitan Kerem, M.D., principal investigator and head of the Department of Pediatrics and Cystic Fibrosis Center. Separately, results from a European study evaluating 14-day courses of PTC124 in pediatric patients with nonsense-mutation-mediated CF confirmed the CFTR activity observed in previous short-term studies in adult patients. Data from the European study were presented today by Isabelle Sermet-Gaudelus, M.D., Ph.D., principal investigator at l'H˘pital Necker-Enfants Malade.
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<br />Patients with CF lack adequate levels of the CFTR protein, a chloride channel that maintains proper hydration of epithelial surfaces in the lung, pancreas, and liver. Patients with nonsense-mutation-mediated CF generally have a severe form of CF because virtually no CFTR protein is produced. Previous studies of PTC124 adult patients with CF evaluated nasal transepithelial potential difference (TEPD) as a surrogate for the presence and activity of the CFTR protein. Across the short- and long-term clinical trials at high and low doses of PTC124, TEPD assessments showed statistically significant improvements of mean CFTR-dependent chloride secretion in the airways.
<br />
<br />The Phase 2a extension study in Israel assessed 3 months of oral PTC124 therapy at two different dose levels in 19 adult men and women with nonsense-mutation-mediated CF who had participated in a prior short-term PTC124 Phase 2a study. More than 90% of the patients had chronic CF-related lung infection and also had CF-induced pancreatic insufficiency. Results from the study showed that treatment with PTC124 resulted in statistically significant (p<0.001) improvements in CFTR function as measured by TEPD in both dose groups. The proportion of patients showing improvement in TEPD chloride secretion increased over time in the extension study. Trends towards improvements in mean FEV1 and FVC values were observed. Baseline data showed that CF patients cough a remarkable 643 times per day on average, with a range of 324 to 1,569 coughs per day. In comparison, healthy individuals generally cough fewer than 16 times per day, according to the European Respiratory Journal (Hsu 1994). Patients receiving PTC124 experienced a mean [28%] decrease in cough frequency by the end of three months of therapy (p<0.01). PTC124 was generally well tolerated, resulting in excellent mean compliance with the treatment regimen (>90%).
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<br />"Three months of treatment with PTC124 in nonsense-mutation-mediated CF patients was associated with time-dependent improvements in nasal TEPD chloride conductance, pulmonary function and cough - important markers suggesting the potential for benefit in patients with CF," stated Dr. Wilschanski. "PTC124 increases CFTR-mediated chloride secretion in patients with a variety of nonsense mutation types, which suggests a broad spectrum of activity across one of the major subpopulations in CF."
<br />
<br />Dr. Kerem added, "The impact on cough that we observed in this study by objective measurement is particularly notable given that cough is one of the major symptomatic manifestations of the underlying disease process in CF. The reduction in cough achieved with PTC124 in this three-month study suggests secondary clinical effects of drug activity. Based on these findings, we believe that objective measurement of cough may offer a meaningful new way to evaluate drug efficacy in future CF clinical trials."
<br />
<br />In a separate Phase 2a study in Europe, data are currently available from 21 children who received two 14-day treatment courses of oral PTC124 therapy at two different dose levels. Patients ranged in age from six to 18 years. All had nonsense-mutation-mediated disease, pathological lung infection, and pancreatic insufficiency. Statistically significant (p<0.05) increases in the proportion of epithelial cells showing surface staining with the CFTR protein were observed. In addition, statistically significant (p<0.05) improvements in CFTR-mediated chloride conductance as measured by TEPD were evident. PTC124 was generally well tolerated in pediatric patients and mean compliance with treatment was excellent (>95%).
<br />
<br />"We are encouraged to see the activity of PTC124 observed in adults reproduced in a pediatric population in France and Belgium," commented Dr. Sermet-Gaudelus. "PTC124 causes the missing protein to be made, to be located in the right place in the cell, and to have functional effect. Combined with the generally well-tolerated profile of PTC124, we believe these data support inclusion of pediatric patients in future clinical trials."
<br />
<br />"We are gratified by these results from our Israeli, French, and Belgian investigators, which demonstrated the activity of PTC124 in patients across a variety of age ranges, geographies, and nonsense mutation types," stated Langdon Miller, M.D., Chief Medical Officer of PTC. "These data offer the basis for initiating a randomized, controlled Phase 2b study later this year to evaluate the clinical benefit of PTC124 in adults and children with nonsense-mutation-mediated CF."
<br />
<br />ABOUT CYSTIC FIBROSIS
<br />Cystic fibrosis (CF) is among the most common life-threatening genetic disorders worldwide. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and, according to the European Cystic Fibrosis Foundation, it affects a similar number of patients in Europe. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10% of patients in the United States. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion. There is a significant unmet medical need for treatments that address the underlying cause of CF. More information regarding CF is available through the Cystic Fibrosis Foundation (www.cff.org).
<br />
<br />ABOUT PTC124
<br />PTC124 is an orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce functional protein across a variety of nonsense mutation types. Across all clinical studies to date, PTC124 has been generally well tolerated and has achieved target plasma concentrations that have been associated with activity in preclinical models. PTC124 is currently in Phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mediated genetic disorders may provide clinical benefits.
<br />
<br />ABOUT PTC THERAPEUTICS INC.
<br />PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-molecules (GEMS) technology platform, which has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Pfizer, Celgene, CV Therapeutics and Schering-Plough. For more information, visit the company's website, www.ptcbio.com.