VX-770 Update

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Mommafirst

Guest
I went to a talk last week where Dr. Beall (the pres. of CFF) spoke. He discussed exactly what you are saying and it is very very exciting. One pill, once a day and anyone with those mutuations could have SIGNIFICANT help with their CF. Trials thus far are showing an average of 12% PFT increases in just the first 14 days!!!!

My daughter doesn't have either of the mutations that VX-770 and VX-809 help, but I believe that this is the first step toward a more global understanding how to make a huge difference in CF care.

Atalauren (for those mutations with an X at the end) is not doing as well in clinical trials. : ( For some reason it has had great outcomes in Israel trials, but not so great in the US. I hope that this one can yield better results -- my daughter has an X mutation.
 
M

Mommafirst

Guest
I went to a talk last week where Dr. Beall (the pres. of CFF) spoke. He discussed exactly what you are saying and it is very very exciting. One pill, once a day and anyone with those mutuations could have SIGNIFICANT help with their CF. Trials thus far are showing an average of 12% PFT increases in just the first 14 days!!!!

My daughter doesn't have either of the mutations that VX-770 and VX-809 help, but I believe that this is the first step toward a more global understanding how to make a huge difference in CF care.

Atalauren (for those mutations with an X at the end) is not doing as well in clinical trials. : ( For some reason it has had great outcomes in Israel trials, but not so great in the US. I hope that this one can yield better results -- my daughter has an X mutation.
 
M

Mommafirst

Guest
I went to a talk last week where Dr. Beall (the pres. of CFF) spoke. He discussed exactly what you are saying and it is very very exciting. One pill, once a day and anyone with those mutuations could have SIGNIFICANT help with their CF. Trials thus far are showing an average of 12% PFT increases in just the first 14 days!!!!

My daughter doesn't have either of the mutations that VX-770 and VX-809 help, but I believe that this is the first step toward a more global understanding how to make a huge difference in CF care.

Atalauren (for those mutations with an X at the end) is not doing as well in clinical trials. : ( For some reason it has had great outcomes in Israel trials, but not so great in the US. I hope that this one can yield better results -- my daughter has an X mutation.
 
M

Mommafirst

Guest
I went to a talk last week where Dr. Beall (the pres. of CFF) spoke. He discussed exactly what you are saying and it is very very exciting. One pill, once a day and anyone with those mutuations could have SIGNIFICANT help with their CF. Trials thus far are showing an average of 12% PFT increases in just the first 14 days!!!!

My daughter doesn't have either of the mutations that VX-770 and VX-809 help, but I believe that this is the first step toward a more global understanding how to make a huge difference in CF care.

Atalauren (for those mutations with an X at the end) is not doing as well in clinical trials. : ( For some reason it has had great outcomes in Israel trials, but not so great in the US. I hope that this one can yield better results -- my daughter has an X mutation.
 
M

Mommafirst

Guest
I went to a talk last week where Dr. Beall (the pres. of CFF) spoke. He discussed exactly what you are saying and it is very very exciting. One pill, once a day and anyone with those mutuations could have SIGNIFICANT help with their CF. Trials thus far are showing an average of 12% PFT increases in just the first 14 days!!!!
<br />
<br />My daughter doesn't have either of the mutations that VX-770 and VX-809 help, but I believe that this is the first step toward a more global understanding how to make a huge difference in CF care.
<br />
<br />Atalauren (for those mutations with an X at the end) is not doing as well in clinical trials. : ( For some reason it has had great outcomes in Israel trials, but not so great in the US. I hope that this one can yield better results -- my daughter has an X mutation.
 

missT

Member
Hi Mommafirst, I have read the outcome of Ataluren and maybe I am just being hopeful but it looks like its working. Ugh, I am happy for anyone who can be cured but I do not have the D508 or the other mutation with the G. I just wish this mucus would stop.
 

missT

Member
Hi Mommafirst, I have read the outcome of Ataluren and maybe I am just being hopeful but it looks like its working. Ugh, I am happy for anyone who can be cured but I do not have the D508 or the other mutation with the G. I just wish this mucus would stop.
 

missT

Member
Hi Mommafirst, I have read the outcome of Ataluren and maybe I am just being hopeful but it looks like its working. Ugh, I am happy for anyone who can be cured but I do not have the D508 or the other mutation with the G. I just wish this mucus would stop.
 

missT

Member
Hi Mommafirst, I have read the outcome of Ataluren and maybe I am just being hopeful but it looks like its working. Ugh, I am happy for anyone who can be cured but I do not have the D508 or the other mutation with the G. I just wish this mucus would stop.
 

missT

Member
Hi Mommafirst, I have read the outcome of Ataluren and maybe I am just being hopeful but it looks like its working. Ugh, I am happy for anyone who can be cured but I do not have the D508 or the other mutation with the G. I just wish this mucus would stop.
 

hopefulmommy

New member
if my son were old enough I'd put him in a trial in a heartbeat!!! Unfortunately he's only 2, but we're so excited about these new drugs!
 

hopefulmommy

New member
if my son were old enough I'd put him in a trial in a heartbeat!!! Unfortunately he's only 2, but we're so excited about these new drugs!
 

hopefulmommy

New member
if my son were old enough I'd put him in a trial in a heartbeat!!! Unfortunately he's only 2, but we're so excited about these new drugs!
 

hopefulmommy

New member
if my son were old enough I'd put him in a trial in a heartbeat!!! Unfortunately he's only 2, but we're so excited about these new drugs!
 

hopefulmommy

New member
if my son were old enough I'd put him in a trial in a heartbeat!!! Unfortunately he's only 2, but we're so excited about these new drugs!
 

LouLou

New member
I was in Ph. 2B and am now on Day 3 of Ph. 3 (a 48 week study). I'm not allowed to discuss my trial experience on the web but I just wanted to share that I'm excited to once again be in this trial.
 

LouLou

New member
I was in Ph. 2B and am now on Day 3 of Ph. 3 (a 48 week study). I'm not allowed to discuss my trial experience on the web but I just wanted to share that I'm excited to once again be in this trial.
 

LouLou

New member
I was in Ph. 2B and am now on Day 3 of Ph. 3 (a 48 week study). I'm not allowed to discuss my trial experience on the web but I just wanted to share that I'm excited to once again be in this trial.
 

LouLou

New member
I was in Ph. 2B and am now on Day 3 of Ph. 3 (a 48 week study). I'm not allowed to discuss my trial experience on the web but I just wanted to share that I'm excited to once again be in this trial.
 

LouLou

New member
I was in Ph. 2B and am now on Day 3 of Ph. 3 (a 48 week study). I'm not allowed to discuss my trial experience on the web but I just wanted to share that I'm excited to once again be in this trial.
 
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