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annalisa
02-10-2012, 01:48 PM
Hi, nobody has got r347p mutation (class iv)?
does vertex think that vertex770 will work also for it??
i am a mum, and i really nedd a hope...
thanl you to everybody, stay healty!

annalisa
02-10-2012, 01:48 PM
Hi, nobody has got r347p mutation (class iv)?
does vertex think that vertex770 will work also for it??
i am a mum, and i really nedd a hope...
thanl you to everybody, stay healty!

marcijo
02-10-2012, 02:15 PM
I have R117H which is a class 4 - and I know Vertex is going to start a study soon on people with at least one R117H mutation....I believe they are thinking it will work with the R117H (and hopefully that means the rest of the class 4 mutations).

marcijo
02-10-2012, 02:15 PM
I have R117H which is a class 4 - and I know Vertex is going to start a study soon on people with at least one R117H mutation....I believe they are thinking it will work with the R117H (and hopefully that means the rest of the class 4 mutations).

annalisa
02-13-2012, 07:12 AM
thank you marci!
good luck for all of us!!!!!!

annalisa
02-13-2012, 07:12 AM
thank you marci!
good luck for all of us!!!!!!

ffevziyildiz
02-13-2012, 12:23 PM
I m from Turkey and i have a son who has got F508R347p mutations. I hope Vertex vx-770 (Kalydeco) will work in our mutations. Annalisa can we meet about our mutations?

ffevziyildiz
02-13-2012, 12:23 PM
I m from Turkey and i have a son who has got F508R347p mutations. I hope Vertex vx-770 (Kalydeco) will work in our mutations. Annalisa can we meet about our mutations?

annalisa
02-13-2012, 01:34 PM
hi ffevziyildiz, my daugther has got a rare mutation belonging to class I (but not a "stop one") and r347p
the doctors told us that we can hope in a "mild" case, due to r347p, and I really hope so...Mu dautgher is 2 years old, and she is doing well, at the moment (the only problem is the pancreas, that (only) sometimes seems not to work properly).
how old is your son?
how is he going on?

annalisa
02-13-2012, 01:34 PM
hi ffevziyildiz, my daugther has got a rare mutation belonging to class I (but not a "stop one") and r347p
the doctors told us that we can hope in a "mild" case, due to r347p, and I really hope so...Mu dautgher is 2 years old, and she is doing well, at the moment (the only problem is the pancreas, that (only) sometimes seems not to work properly).
how old is your son?
how is he going on?

Jenica
02-13-2012, 02:41 PM
My daughter is DF508 and R347P, and I don't believe Kalydeco would be effective for her, but they are in the middle of Phase II trials combining vx770 and vx809 to target the DF508 mutation.

ffevziyildiz, where in Turkey are you? Friends of ours moved there about nine years ago.

Jenica
02-13-2012, 02:41 PM
My daughter is DF508 and R347P, and I don't believe Kalydeco would be effective for her, but they are in the middle of Phase II trials combining vx770 and vx809 to target the DF508 mutation.

ffevziyildiz, where in Turkey are you? Friends of ours moved there about nine years ago.

annalisa
02-13-2012, 02:46 PM
hy jenica,
could you please tell me if your daughter is pancreatic sufficient?

annalisa
02-13-2012, 02:46 PM
hy jenica,
could you please tell me if your daughter is pancreatic sufficient?

Jenica
02-13-2012, 04:10 PM
Annalisa, she is not. I know that her mutation suggests that she SHOULD be, but she isn't. Her sweat test also came back through the roof, and that was also a bit unexpected for her R347P mutation. Are you pancreatic sufficient?

We attended the Annual CF dinner on Saturday night and Dr. Beall spoke about their efforts at increasing the percentage of patients benefiting from Kalydeco from 4% to 10, 15, even 20%. He didn't mention which mutations they're targeting for this, but I'm wondering if it might be some Class IVs, since the protein for them is already in the correct place.

I was so encouraged listening to him speak, because they are NOT just waiting to see if the combined vx-809 with Kalydeco will work with DF508 ... they are still aggressively pursuing many different compounds. It is nice to know that they have many things in the pipeline and they aren't resting solely on the vx-809/Kalydeco study. That said, hopefully that one will be successful and we won't need further compounds! <img src="i/expressions/face-icon-small-smile.gif" border="0">

Jenica
02-13-2012, 04:10 PM
Annalisa, she is not. I know that her mutation suggests that she SHOULD be, but she isn't. Her sweat test also came back through the roof, and that was also a bit unexpected for her R347P mutation. Are you pancreatic sufficient?

We attended the Annual CF dinner on Saturday night and Dr. Beall spoke about their efforts at increasing the percentage of patients benefiting from Kalydeco from 4% to 10, 15, even 20%. He didn't mention which mutations they're targeting for this, but I'm wondering if it might be some Class IVs, since the protein for them is already in the correct place.

I was so encouraged listening to him speak, because they are NOT just waiting to see if the combined vx-809 with Kalydeco will work with DF508 ... they are still aggressively pursuing many different compounds. It is nice to know that they have many things in the pipeline and they aren't resting solely on the vx-809/Kalydeco study. That said, hopefully that one will be successful and we won't need further compounds! <img src="i/expressions/face-icon-small-smile.gif" border="0">

mom2JD
02-14-2012, 03:09 PM
My son has R347P / DF508 and he is completely pancreatic sufficient.

Jenica: thanks for sharing this. I am also still hoping. But R117H has also a gating problem (reduced channel open time) so that this would be different from other class 4 mutations.

mom2JD
02-14-2012, 03:09 PM
My son has R347P / DF508 and he is completely pancreatic sufficient.

Jenica: thanks for sharing this. I am also still hoping. But R117H has also a gating problem (reduced channel open time) so that this would be different from other class 4 mutations.

Gorf
02-14-2012, 08:38 PM
I am 46 year old male who has been on enzymes since diagnosed at 3. (because of no weight gain.) I have 2 DF508, and am anxiously awaiting the Kalydeco and VX-809 trials to be completed.

Gorf
02-14-2012, 08:38 PM
I am 46 year old male who has been on enzymes since diagnosed at 3. (because of no weight gain.) I have 2 DF508, and am anxiously awaiting the Kalydeco and VX-809 trials to be completed.

annalisa
02-21-2012, 07:01 AM
Hi, thank you for your answers!
I red that they think that kalideco can work not onvly on r117h but also on other class iv mutations: at the moment they tested in vitro D1152H and I148T.
so, let's hope..
stay healty

annalisa
02-21-2012, 07:01 AM
Hi, thank you for your answers!
I red that they think that kalideco can work not onvly on r117h but also on other class iv mutations: at the moment they tested in vitro D1152H and I148T.
so, let's hope..
stay healty