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LeftY
08-05-2007, 11:23 PM
does someone have more news about that pill?

LeftY
08-05-2007, 11:23 PM
does someone have more news about that pill?

LeftY
08-05-2007, 11:23 PM
does someone have more news about that pill?

LeftY
08-05-2007, 11:23 PM
does someone have more news about that pill?

LeftY
08-05-2007, 11:23 PM
does someone have more news about that pill?

princessjdc
08-05-2007, 11:52 PM
I havent last I heard it was in trial 2 or 3. I cant wait for it to come out though.

princessjdc
08-05-2007, 11:52 PM
I havent last I heard it was in trial 2 or 3. I cant wait for it to come out though.

princessjdc
08-05-2007, 11:52 PM
I havent last I heard it was in trial 2 or 3. I cant wait for it to come out though.

princessjdc
08-05-2007, 11:52 PM
I havent last I heard it was in trial 2 or 3. I cant wait for it to come out though.

princessjdc
08-05-2007, 11:52 PM
I havent last I heard it was in trial 2 or 3. I cant wait for it to come out though.

LeftY
08-06-2007, 09:09 AM
i read that he pass 2 trials, and now is in final stage...
it will be out in about 2 years. (2009 is d-day <img src="i/expressions/face-icon-small-smile.gif" border="0"> )

LeftY
08-06-2007, 09:09 AM
i read that he pass 2 trials, and now is in final stage...
it will be out in about 2 years. (2009 is d-day <img src="i/expressions/face-icon-small-smile.gif" border="0"> )

LeftY
08-06-2007, 09:09 AM
i read that he pass 2 trials, and now is in final stage...
it will be out in about 2 years. (2009 is d-day <img src="i/expressions/face-icon-small-smile.gif" border="0"> )

LeftY
08-06-2007, 09:09 AM
i read that he pass 2 trials, and now is in final stage...
it will be out in about 2 years. (2009 is d-day <img src="i/expressions/face-icon-small-smile.gif" border="0"> )

LeftY
08-06-2007, 09:09 AM
i read that he pass 2 trials, and now is in final stage...
it will be out in about 2 years. (2009 is d-day <img src="i/expressions/face-icon-small-smile.gif" border="0"> )

zoeg
08-06-2007, 10:48 AM
Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!

Zoe Mom to Kate wCF

zoeg
08-06-2007, 10:48 AM
Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!

Zoe Mom to Kate wCF

zoeg
08-06-2007, 10:48 AM
Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!

Zoe Mom to Kate wCF

zoeg
08-06-2007, 10:48 AM
Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!

Zoe Mom to Kate wCF

zoeg
08-06-2007, 10:48 AM
Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!

Zoe Mom to Kate wCF

blkmsmommy
08-06-2007, 11:35 AM
what is the pill for and where can i get more info on it

blkmsmommy
08-06-2007, 11:35 AM
what is the pill for and where can i get more info on it

blkmsmommy
08-06-2007, 11:35 AM
what is the pill for and where can i get more info on it

blkmsmommy
08-06-2007, 11:35 AM
what is the pill for and where can i get more info on it

blkmsmommy
08-06-2007, 11:35 AM
what is the pill for and where can i get more info on it

dyza
08-06-2007, 02:24 PM
if memory serves me correctly, the pill is specifically towards nonsense mutations, these are mutations that end in an X I believe

<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00234663">http://www.clinicaltrials.gov/ct/show/NCT00234663</a>

dyza
08-06-2007, 02:24 PM
if memory serves me correctly, the pill is specifically towards nonsense mutations, these are mutations that end in an X I believe

<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00234663">http://www.clinicaltrials.gov/ct/show/NCT00234663</a>

dyza
08-06-2007, 02:24 PM
if memory serves me correctly, the pill is specifically towards nonsense mutations, these are mutations that end in an X I believe

<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00234663">http://www.clinicaltrials.gov/ct/show/NCT00234663</a>

dyza
08-06-2007, 02:24 PM
if memory serves me correctly, the pill is specifically towards nonsense mutations, these are mutations that end in an X I believe

<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00234663">http://www.clinicaltrials.gov/ct/show/NCT00234663</a>

dyza
08-06-2007, 02:24 PM
if memory serves me correctly, the pill is specifically towards nonsense mutations, these are mutations that end in an X I believe

<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00234663">http://www.clinicaltrials.gov/ct/show/NCT00234663</a>

rarab43
08-06-2007, 03:04 PM
My son was in the last phase of the study and I asked his drs about what was happening with the next phase and they said the FDA had ordered some more toxicity studies before the next phase. Susan

rarab43
08-06-2007, 03:04 PM
My son was in the last phase of the study and I asked his drs about what was happening with the next phase and they said the FDA had ordered some more toxicity studies before the next phase. Susan

rarab43
08-06-2007, 03:04 PM
My son was in the last phase of the study and I asked his drs about what was happening with the next phase and they said the FDA had ordered some more toxicity studies before the next phase. Susan

rarab43
08-06-2007, 03:04 PM
My son was in the last phase of the study and I asked his drs about what was happening with the next phase and they said the FDA had ordered some more toxicity studies before the next phase. Susan

rarab43
08-06-2007, 03:04 PM
My son was in the last phase of the study and I asked his drs about what was happening with the next phase and they said the FDA had ordered some more toxicity studies before the next phase. Susan

LeftY
08-06-2007, 03:24 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>zoeg</b></i>

Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!



Zoe Mom to Kate wCF</end quote></div>

no problem!
im glad that this info helped you <img src="i/expressions/face-icon-small-smile.gif" border="0">


@rarab43:
your son is testing this pill(s)?

LeftY
08-06-2007, 03:24 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>zoeg</b></i>

Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!



Zoe Mom to Kate wCF</end quote></div>

no problem!
im glad that this info helped you <img src="i/expressions/face-icon-small-smile.gif" border="0">


@rarab43:
your son is testing this pill(s)?

LeftY
08-06-2007, 03:24 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>zoeg</b></i>

Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!



Zoe Mom to Kate wCF</end quote></div>

no problem!
im glad that this info helped you <img src="i/expressions/face-icon-small-smile.gif" border="0">


@rarab43:
your son is testing this pill(s)?

LeftY
08-06-2007, 03:24 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>zoeg</b></i>

Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!



Zoe Mom to Kate wCF</end quote>

no problem!
im glad that this info helped you <img src="i/expressions/face-icon-small-smile.gif" border="0">


@rarab43:
your son is testing this pill(s)?

LeftY
08-06-2007, 03:24 PM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>zoeg</b></i>

Woo Hoo! I have a daughter with a nonsense mutation and I cannot wait for this to actually come out! thanks for the info!



Zoe Mom to Kate wCF</end quote>

no problem!
im glad that this info helped you <img src="i/expressions/face-icon-small-smile.gif" border="0">


@rarab43:
your son is testing this pill(s)?

janddburke
08-06-2007, 09:39 PM
more recent update to the link provided above
<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00458341?order=8
">http://www.clinicaltrials.gov/...w/NCT00458341?order=8
</a>
we too are axiously awaiting availability of this new drug.

--Debbie

janddburke
08-06-2007, 09:39 PM
more recent update to the link provided above
<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00458341?order=8
">http://www.clinicaltrials.gov/...w/NCT00458341?order=8
</a>
we too are axiously awaiting availability of this new drug.

--Debbie

janddburke
08-06-2007, 09:39 PM
more recent update to the link provided above
<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00458341?order=8
">http://www.clinicaltrials.gov/...w/NCT00458341?order=8
</a>
we too are axiously awaiting availability of this new drug.

--Debbie

janddburke
08-06-2007, 09:39 PM
more recent update to the link provided above
<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00458341?order=8
">http://www.clinicaltrials.gov/...w/NCT00458341?order=8
</a>
we too are axiously awaiting availability of this new drug.

--Debbie

janddburke
08-06-2007, 09:39 PM
more recent update to the link provided above
<a target=_blank class=ftalternatingbarlinklarge href="http://www.clinicaltrials.gov/ct/show/NCT00458341?order=8
">http://www.clinicaltrials.gov/...w/NCT00458341?order=8
</a>
we too are axiously awaiting availability of this new drug.

--Debbie

hardclubber
08-13-2007, 02:42 PM
Hi all PTC124 followers. A very good friend just send me the abstracts of the European CF conference in Belek, Turkey this year where results of PTC124 were presented. Below the abstract, I was very happy to see that also mutations with single X and delF508 were included and not only double X mutations. Encouraging news. If anyone else finds some other materials please share here!!
Regards
Jeanpaul

"PTC124 activity in CF pts carrying stop mutations: results of a phase 2 study" E. Kerem et al., various hospitals in Israel

Aim: PTC 124 is a molecule that promotes ribosomal read-through of nonsense mutations in mRNA. The pharmacological activity of PTC124 was determined in CF pts carrying stop mutations. Methods: Pts receiving 2 cycles of oral PTC124 comprising 3 times per say (TID) dosing at 4,4, and 8 mg/kg for 14 days, then no treatment for 14 days, then
TID dosing at 10, 10, and 20 mg/kg for 14 days, and no treatment for 14 days. Nasal potential difference (NPD), pulmonary function, weight and CF symptoms were assessed before and at the end of treatment in each cycle. Results: 23 pts, mean age 25, range 18-57 years, genotypes included: G542X/W1282X; G542X.delF508, G542X/N1303K, W1282X.W1282Xl W1282X/3849+10C to T; 3849+10C to T/delF508; W1282X.delF508 received both dose levels. At both doses, mean chloride conductance (avg of the 2 nostrils) improved from +1.1 to -5.8mV (p<.0001), and from -0.3 to -3.9mV (p=0.032), and normalized to >-5mV in 56% (13/23) and 35% (8/23) of patients, respectively. At the end of low-dose treatment, mean FEV1 increased from 63.6 to 66.7% of normal (p=0.02), FVC increased from 77.0 to 80.1% of normal (p=0.01) and mean body weight increased from 57.0 to 57.7 kg (p<0.001). Side effects were minimal. Conclusions: Treatment with PTC124 in CT pts carrying stop mutations is associated with improvements in NPD, pulmonary functions, and weight. Longer term studies are in progress.

hardclubber
08-13-2007, 02:42 PM
Hi all PTC124 followers. A very good friend just send me the abstracts of the European CF conference in Belek, Turkey this year where results of PTC124 were presented. Below the abstract, I was very happy to see that also mutations with single X and delF508 were included and not only double X mutations. Encouraging news. If anyone else finds some other materials please share here!!
Regards
Jeanpaul

"PTC124 activity in CF pts carrying stop mutations: results of a phase 2 study" E. Kerem et al., various hospitals in Israel

Aim: PTC 124 is a molecule that promotes ribosomal read-through of nonsense mutations in mRNA. The pharmacological activity of PTC124 was determined in CF pts carrying stop mutations. Methods: Pts receiving 2 cycles of oral PTC124 comprising 3 times per say (TID) dosing at 4,4, and 8 mg/kg for 14 days, then no treatment for 14 days, then
TID dosing at 10, 10, and 20 mg/kg for 14 days, and no treatment for 14 days. Nasal potential difference (NPD), pulmonary function, weight and CF symptoms were assessed before and at the end of treatment in each cycle. Results: 23 pts, mean age 25, range 18-57 years, genotypes included: G542X/W1282X; G542X.delF508, G542X/N1303K, W1282X.W1282Xl W1282X/3849+10C to T; 3849+10C to T/delF508; W1282X.delF508 received both dose levels. At both doses, mean chloride conductance (avg of the 2 nostrils) improved from +1.1 to -5.8mV (p<.0001), and from -0.3 to -3.9mV (p=0.032), and normalized to >-5mV in 56% (13/23) and 35% (8/23) of patients, respectively. At the end of low-dose treatment, mean FEV1 increased from 63.6 to 66.7% of normal (p=0.02), FVC increased from 77.0 to 80.1% of normal (p=0.01) and mean body weight increased from 57.0 to 57.7 kg (p<0.001). Side effects were minimal. Conclusions: Treatment with PTC124 in CT pts carrying stop mutations is associated with improvements in NPD, pulmonary functions, and weight. Longer term studies are in progress.

hardclubber
08-13-2007, 02:42 PM
Hi all PTC124 followers. A very good friend just send me the abstracts of the European CF conference in Belek, Turkey this year where results of PTC124 were presented. Below the abstract, I was very happy to see that also mutations with single X and delF508 were included and not only double X mutations. Encouraging news. If anyone else finds some other materials please share here!!
Regards
Jeanpaul

"PTC124 activity in CF pts carrying stop mutations: results of a phase 2 study" E. Kerem et al., various hospitals in Israel

Aim: PTC 124 is a molecule that promotes ribosomal read-through of nonsense mutations in mRNA. The pharmacological activity of PTC124 was determined in CF pts carrying stop mutations. Methods: Pts receiving 2 cycles of oral PTC124 comprising 3 times per say (TID) dosing at 4,4, and 8 mg/kg for 14 days, then no treatment for 14 days, then
TID dosing at 10, 10, and 20 mg/kg for 14 days, and no treatment for 14 days. Nasal potential difference (NPD), pulmonary function, weight and CF symptoms were assessed before and at the end of treatment in each cycle. Results: 23 pts, mean age 25, range 18-57 years, genotypes included: G542X/W1282X; G542X.delF508, G542X/N1303K, W1282X.W1282Xl W1282X/3849+10C to T; 3849+10C to T/delF508; W1282X.delF508 received both dose levels. At both doses, mean chloride conductance (avg of the 2 nostrils) improved from +1.1 to -5.8mV (p<.0001), and from -0.3 to -3.9mV (p=0.032), and normalized to >-5mV in 56% (13/23) and 35% (8/23) of patients, respectively. At the end of low-dose treatment, mean FEV1 increased from 63.6 to 66.7% of normal (p=0.02), FVC increased from 77.0 to 80.1% of normal (p=0.01) and mean body weight increased from 57.0 to 57.7 kg (p<0.001). Side effects were minimal. Conclusions: Treatment with PTC124 in CT pts carrying stop mutations is associated with improvements in NPD, pulmonary functions, and weight. Longer term studies are in progress.

hardclubber
08-13-2007, 02:42 PM
Hi all PTC124 followers. A very good friend just send me the abstracts of the European CF conference in Belek, Turkey this year where results of PTC124 were presented. Below the abstract, I was very happy to see that also mutations with single X and delF508 were included and not only double X mutations. Encouraging news. If anyone else finds some other materials please share here!!
Regards
Jeanpaul

"PTC124 activity in CF pts carrying stop mutations: results of a phase 2 study" E. Kerem et al., various hospitals in Israel

Aim: PTC 124 is a molecule that promotes ribosomal read-through of nonsense mutations in mRNA. The pharmacological activity of PTC124 was determined in CF pts carrying stop mutations. Methods: Pts receiving 2 cycles of oral PTC124 comprising 3 times per say (TID) dosing at 4,4, and 8 mg/kg for 14 days, then no treatment for 14 days, then
TID dosing at 10, 10, and 20 mg/kg for 14 days, and no treatment for 14 days. Nasal potential difference (NPD), pulmonary function, weight and CF symptoms were assessed before and at the end of treatment in each cycle. Results: 23 pts, mean age 25, range 18-57 years, genotypes included: G542X/W1282X; G542X.delF508, G542X/N1303K, W1282X.W1282Xl W1282X/3849+10C to T; 3849+10C to T/delF508; W1282X.delF508 received both dose levels. At both doses, mean chloride conductance (avg of the 2 nostrils) improved from +1.1 to -5.8mV (p<.0001), and from -0.3 to -3.9mV (p=0.032), and normalized to >-5mV in 56% (13/23) and 35% (8/23) of patients, respectively. At the end of low-dose treatment, mean FEV1 increased from 63.6 to 66.7% of normal (p=0.02), FVC increased from 77.0 to 80.1% of normal (p=0.01) and mean body weight increased from 57.0 to 57.7 kg (p<0.001). Side effects were minimal. Conclusions: Treatment with PTC124 in CT pts carrying stop mutations is associated with improvements in NPD, pulmonary functions, and weight. Longer term studies are in progress.

hardclubber
08-13-2007, 02:42 PM
Hi all PTC124 followers. A very good friend just send me the abstracts of the European CF conference in Belek, Turkey this year where results of PTC124 were presented. Below the abstract, I was very happy to see that also mutations with single X and delF508 were included and not only double X mutations. Encouraging news. If anyone else finds some other materials please share here!!
Regards
Jeanpaul

"PTC124 activity in CF pts carrying stop mutations: results of a phase 2 study" E. Kerem et al., various hospitals in Israel

Aim: PTC 124 is a molecule that promotes ribosomal read-through of nonsense mutations in mRNA. The pharmacological activity of PTC124 was determined in CF pts carrying stop mutations. Methods: Pts receiving 2 cycles of oral PTC124 comprising 3 times per say (TID) dosing at 4,4, and 8 mg/kg for 14 days, then no treatment for 14 days, then
TID dosing at 10, 10, and 20 mg/kg for 14 days, and no treatment for 14 days. Nasal potential difference (NPD), pulmonary function, weight and CF symptoms were assessed before and at the end of treatment in each cycle. Results: 23 pts, mean age 25, range 18-57 years, genotypes included: G542X/W1282X; G542X.delF508, G542X/N1303K, W1282X.W1282Xl W1282X/3849+10C to T; 3849+10C to T/delF508; W1282X.delF508 received both dose levels. At both doses, mean chloride conductance (avg of the 2 nostrils) improved from +1.1 to -5.8mV (p<.0001), and from -0.3 to -3.9mV (p=0.032), and normalized to >-5mV in 56% (13/23) and 35% (8/23) of patients, respectively. At the end of low-dose treatment, mean FEV1 increased from 63.6 to 66.7% of normal (p=0.02), FVC increased from 77.0 to 80.1% of normal (p=0.01) and mean body weight increased from 57.0 to 57.7 kg (p<0.001). Side effects were minimal. Conclusions: Treatment with PTC124 in CT pts carrying stop mutations is associated with improvements in NPD, pulmonary functions, and weight. Longer term studies are in progress.

LeftY
08-14-2007, 04:32 AM
sounds like we have... a cure?

LeftY
08-14-2007, 04:32 AM
sounds like we have... a cure?

LeftY
08-14-2007, 04:32 AM
sounds like we have... a cure?

LeftY
08-14-2007, 04:32 AM
sounds like we have... a cure?

LeftY
08-14-2007, 04:32 AM
sounds like we have... a cure?

bittyhorse23
08-14-2007, 06:57 PM
This thread got me interested so I did some research and surprise I am still confused! <img src="i/expressions/face-icon-small-smile.gif" border="0">

What I gathered from my research and reading here is you have to have the nonsense mutation? So people like me with the DDF508 won't benefit from it at all? Does anyone know of anything similar for us non-nonsense patients?

It does sound like a possible cure though.....very interesting!!!!!!!!!!!!!!! I can't wait till it comes out (even if I can't benefit from it!).

bittyhorse23
08-14-2007, 06:57 PM
This thread got me interested so I did some research and surprise I am still confused! <img src="i/expressions/face-icon-small-smile.gif" border="0">

What I gathered from my research and reading here is you have to have the nonsense mutation? So people like me with the DDF508 won't benefit from it at all? Does anyone know of anything similar for us non-nonsense patients?

It does sound like a possible cure though.....very interesting!!!!!!!!!!!!!!! I can't wait till it comes out (even if I can't benefit from it!).

bittyhorse23
08-14-2007, 06:57 PM
This thread got me interested so I did some research and surprise I am still confused! <img src="i/expressions/face-icon-small-smile.gif" border="0">

What I gathered from my research and reading here is you have to have the nonsense mutation? So people like me with the DDF508 won't benefit from it at all? Does anyone know of anything similar for us non-nonsense patients?

It does sound like a possible cure though.....very interesting!!!!!!!!!!!!!!! I can't wait till it comes out (even if I can't benefit from it!).

bittyhorse23
08-14-2007, 06:57 PM
This thread got me interested so I did some research and surprise I am still confused! <img src="i/expressions/face-icon-small-smile.gif" border="0">

What I gathered from my research and reading here is you have to have the nonsense mutation? So people like me with the DDF508 won't benefit from it at all? Does anyone know of anything similar for us non-nonsense patients?

It does sound like a possible cure though.....very interesting!!!!!!!!!!!!!!! I can't wait till it comes out (even if I can't benefit from it!).

bittyhorse23
08-14-2007, 06:57 PM
This thread got me interested so I did some research and surprise I am still confused! <img src="i/expressions/face-icon-small-smile.gif" border="0">

What I gathered from my research and reading here is you have to have the nonsense mutation? So people like me with the DDF508 won't benefit from it at all? Does anyone know of anything similar for us non-nonsense patients?

It does sound like a possible cure though.....very interesting!!!!!!!!!!!!!!! I can't wait till it comes out (even if I can't benefit from it!).

princessjdc
08-14-2007, 07:07 PM
Well not exactly a "cure" but it will help eliminate some of the effects of CF to an extent. With PTC124, I feel they are possibly getting a lot closer to a cure. From what I know of I dont believe they have anything in trial or out for other mutations besides the nonsense one which is in trial right now, sorry, but Im sure that they are in the making of trying to invent a med for the other mutations, give it time and you will start to see other trials going on for other mutations.

princessjdc
08-14-2007, 07:07 PM
Well not exactly a "cure" but it will help eliminate some of the effects of CF to an extent. With PTC124, I feel they are possibly getting a lot closer to a cure. From what I know of I dont believe they have anything in trial or out for other mutations besides the nonsense one which is in trial right now, sorry, but Im sure that they are in the making of trying to invent a med for the other mutations, give it time and you will start to see other trials going on for other mutations.

princessjdc
08-14-2007, 07:07 PM
Well not exactly a "cure" but it will help eliminate some of the effects of CF to an extent. With PTC124, I feel they are possibly getting a lot closer to a cure. From what I know of I dont believe they have anything in trial or out for other mutations besides the nonsense one which is in trial right now, sorry, but Im sure that they are in the making of trying to invent a med for the other mutations, give it time and you will start to see other trials going on for other mutations.

princessjdc
08-14-2007, 07:07 PM
Well not exactly a "cure" but it will help eliminate some of the effects of CF to an extent. With PTC124, I feel they are possibly getting a lot closer to a cure. From what I know of I dont believe they have anything in trial or out for other mutations besides the nonsense one which is in trial right now, sorry, but Im sure that they are in the making of trying to invent a med for the other mutations, give it time and you will start to see other trials going on for other mutations.

princessjdc
08-14-2007, 07:07 PM
Well not exactly a "cure" but it will help eliminate some of the effects of CF to an extent. With PTC124, I feel they are possibly getting a lot closer to a cure. From what I know of I dont believe they have anything in trial or out for other mutations besides the nonsense one which is in trial right now, sorry, but Im sure that they are in the making of trying to invent a med for the other mutations, give it time and you will start to see other trials going on for other mutations.