vx770 for some other mutations!

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samaruba

New member
Two days ago I received the message of my life! It seems that VX770 will probably work on some other mutations than G551D as well. Mostly gating mutations. On of which my son has...... <img src="i/expressions/face-icon-small-blush.gif" border="0">)<br>This is copied out of the abstract:



<span style="font-size: 10.000000pt; font-family: 'Times'">These in vitro results showed that VX-770 caused a robust potentiation of mutant CFTR
forms encoded by the CF-causing <span style="font-size: 10.000000pt; font-family: 'Times'; font-style: italic">CFTR <span style="font-size: 10.000000pt; font-family: 'Times'">gating mutations G551D, G178R, G551S, G970R, G1244E, S1255P,
G1349D, S549N, S549R, and S1251N.<br>
<span style="font-size: 10.000000pt; font-family: 'Times'"><br><span style="font-size: 10.000000pt; font-family: 'Times'">Here is the link to the abstract:<br><a href="http://http://nacfcdl.cff.org/Abstracts%20and%20Posters/10_Van%20Goor_Fredrick.pdf" title="abstract" target="">http://nacfcdl.cff.org/Abstracts%20and%20Posters/10_Van%20Goor_Fredrick.pdf</a>
 
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samaruba

New member
Two days ago I received the message of my life! It seems that VX770 will probably work on some other mutations than G551D as well. Mostly gating mutations. On of which my son has...... <img src="i/expressions/face-icon-small-blush.gif" border="0">)<br>This is copied out of the abstract:



<span style="font-size: 10.000000pt; font-family: 'Times'">These in vitro results showed that VX-770 caused a robust potentiation of mutant CFTR
forms encoded by the CF-causing <span style="font-size: 10.000000pt; font-family: 'Times'; font-style: italic">CFTR <span style="font-size: 10.000000pt; font-family: 'Times'">gating mutations G551D, G178R, G551S, G970R, G1244E, S1255P,
G1349D, S549N, S549R, and S1251N.<br>
<span style="font-size: 10.000000pt; font-family: 'Times'"><br><span style="font-size: 10.000000pt; font-family: 'Times'">Here is the link to the abstract:<br><a href="http://http://nacfcdl.cff.org/Abstracts%20and%20Posters/10_Van%20Goor_Fredrick.pdf" title="abstract" target="">http://nacfcdl.cff.org/Abstracts%20and%20Posters/10_Van%20Goor_Fredrick.pdf</a>
 
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SarahProcter

Guest
Thank you for posting this. Great news! The link doesn't work for me - could you confirm that it is correct? I would love to know what they have tested it on that has NOT worked for, as well.<br>Edited to add: OK, the link works for me if it's changed to be like this, let's see if the editor mangles it:<br><a href="http://nacfcdl.cff.org/Abstracts%20and%20Posters/10_Van%20Goor_Fredrick.pdf" style="background-color: rgb(238, 238, 238); ">http://nacfcdl.cff.org/Abstracts%20and%20Posters/10_Van%20Goor_Fredrick.pdf</a>
 
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SarahProcter

Guest
Thank you for posting this. Great news! The link doesn't work for me - could you confirm that it is correct? I would love to know what they have tested it on that has NOT worked for, as well.<br>Edited to add: OK, the link works for me if it's changed to be like this, let's see if the editor mangles it:<br><a href="http://nacfcdl.cff.org/Abstracts%20and%20Posters/10_Van%20Goor_Fredrick.pdf" style="background-color: rgb(238, 238, 238); ">http://nacfcdl.cff.org/Abstracts%20and%20Posters/10_Van%20Goor_Fredrick.pdf</a>
 
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samaruba

New member
Okay, the link is strange... I didn't know...sorry...!
Otherwise just Google: Van Goor S1251N VX770 For me it is the second link that comes up on Google.
It's a PDF file.
I believe it's a poster presentation from the CF conference in Anaheim. So if all else fails, go to that website and search for it. It's the 10th poster presentation. I could post the link, but the same thing will probably happen as what happened before..... weird!
 
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samaruba

New member
Okay, the link is strange... I didn't know...sorry...!
Otherwise just Google: Van Goor S1251N VX770 For me it is the second link that comes up on Google.
It's a PDF file.
I believe it's a poster presentation from the CF conference in Anaheim. So if all else fails, go to that website and search for it. It's the 10th poster presentation. I could post the link, but the same thing will probably happen as what happened before..... weird!
 
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Kristen

New member
Yup! It's great news. I believe that VX-770 is going to be approved for all gating mutations in Europe.

And, this year they are supposed to begin a clinic trail of VX-770 on people with gating mutations other than G551D. In addition they may include some other Class IV and/or Class V mutations.
 
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Kristen

New member
Yup! It's great news. I believe that VX-770 is going to be approved for all gating mutations in Europe.

And, this year they are supposed to begin a clinic trail of VX-770 on people with gating mutations other than G551D. In addition they may include some other Class IV and/or Class V mutations.
 
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semperfiohana

Guest
I am praying for an awesome b-day present!! This drug is for review on April 18th, my b-day is the 19th! It would be the coolest b-day present ever!!!
 
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semperfiohana

Guest
I am praying for an awesome b-day present!! This drug is for review on April 18th, my b-day is the 19th! It would be the coolest b-day present ever!!!
 
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LouLou

New member
Actually Candice I think it may happen sooner. I spoke with the PI (Principal Investigator) at the clinic I go to for my Kalydeco participation and he said at my last check in that he had been at a meeting for the trial the week before and they said they were being reviewed Feb. 28 and that they were hoping to announce approval in March! Also I learned, those of us on open label, will remain on the trial even after approval to continue to be monitored most likely. This is because it is expected that the FDA is likely to request the pateints who have been on it the longest to be followed since the Ph. 3 data is on such a small set of people and for relatively so short.
 
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LouLou

New member
Actually Candice I think it may happen sooner. I spoke with the PI (Principal Investigator) at the clinic I go to for my Kalydeco participation and he said at my last check in that he had been at a meeting for the trial the week before and they said they were being reviewed Feb. 28 and that they were hoping to announce approval in March! Also I learned, those of us on open label, will remain on the trial even after approval to continue to be monitored most likely. This is because it is expected that the FDA is likely to request the pateints who have been on it the longest to be followed since the Ph. 3 data is on such a small set of people and for relatively so short.
 
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sunshine39656

Member
Oh thank you for posting!!! I got tested a couple of weeks ago to find out what mutation I have...no results yet...but I was bummed because I thought that if I didn't have this one or the one in testing that all was lost. So thanks for the info, it brightens my day <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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sunshine39656

Member
Oh thank you for posting!!! I got tested a couple of weeks ago to find out what mutation I have...no results yet...but I was bummed because I thought that if I didn't have this one or the one in testing that all was lost. So thanks for the info, it brightens my day <img src="i/expressions/face-icon-small-smile.gif" border="0">
 
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semperfiohana

Guest
i wasn't in any of the trials so i'm not currently on it, but i am really looking forward to it being approved!!!
 
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semperfiohana

Guest
i wasn't in any of the trials so i'm not currently on it, but i am really looking forward to it being approved!!!
 
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