VX 770-will it help us?

[Kristen]

Thanks for starting this thread, missT <img src="i/expressions/face-icon-small-smile.gif" border="0"> This question has been burning a hole in my brain, but I've been too shy to start a thread on here!

The news sounds very positive, and it makes sense. If "they" didn't think it would work for us, wouldn't they be looking for more drugs? I am curious to see how insurance will respond to using it off-label.

Interesting point about being Irish - I am 75% Irish!

 

[Kristen]

Thanks for starting this thread, missT <img src="i/expressions/face-icon-small-smile.gif" border="0"> This question has been burning a hole in my brain, but I've been too shy to start a thread on here!
<br />
<br />The news sounds very positive, and it makes sense. If "they" didn't think it would work for us, wouldn't they be looking for more drugs? I am curious to see how insurance will respond to using it off-label.
<br />
<br />Interesting point about being Irish - I am 75% Irish!

 

[gunelle]

Hi, can anyone explain to me what this VX 770 is about exactly. Is it a new drug that will help people with the R117 mutation? And if so how? I have R117C and have been told it's very similar to R177H.

Thanks for replies.

Gunhild

 

[gunelle]

Hi, can anyone explain to me what this VX 770 is about exactly. Is it a new drug that will help people with the R117 mutation? And if so how? I have R117C and have been told it's very similar to R177H.

Thanks for replies.

Gunhild

 

[gunelle]

Hi, can anyone explain to me what this VX 770 is about exactly. Is it a new drug that will help people with the R117 mutation? And if so how? I have R117C and have been told it's very similar to R177H.
<br />
<br />Thanks for replies.
<br />
<br />Gunhild

 

[dyza]

very interesting. I have the RH117 (twice infact) and I am scottish, so there is always a good chance that I too have irish roots on either side of my family.

 

[dyza]

very interesting. I have the RH117 (twice infact) and I am scottish, so there is always a good chance that I too have irish roots on either side of my family.

 

[dyza]

very interesting. I have the RH117 (twice infact) and I am scottish, so there is always a good chance that I too have irish roots on either side of my family.

 

[dasjsmum]

the vx770 directly targets g551d (and possibly other mutations). It works, and people taking it are now through the 48 week trial and about three months onto the open label. My son is completely healthy, hasnt been sick since he started in Nov 2009, fev1 hovering around 89% (he's 32 and been in the trial). He uses pulmozyme sometimes...he isnt using Creon...

This question above is asking whether the vx770 will work the same way for other mutations such as the RH117...hope that helps Gunelle <img src="i/expressions/face-icon-small-smile.gif" border="0"> It's VERY VERY exciting, hoping to get to the market by the end of this year, and they're working on another for df508 which is just starting initial ph 2 (the vx809) <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[dasjsmum]

the vx770 directly targets g551d (and possibly other mutations). It works, and people taking it are now through the 48 week trial and about three months onto the open label. My son is completely healthy, hasnt been sick since he started in Nov 2009, fev1 hovering around 89% (he's 32 and been in the trial). He uses pulmozyme sometimes...he isnt using Creon...

This question above is asking whether the vx770 will work the same way for other mutations such as the RH117...hope that helps Gunelle <img src="i/expressions/face-icon-small-smile.gif" border="0"> It's VERY VERY exciting, hoping to get to the market by the end of this year, and they're working on another for df508 which is just starting initial ph 2 (the vx809) <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[dasjsmum]

the vx770 directly targets g551d (and possibly other mutations). It works, and people taking it are now through the 48 week trial and about three months onto the open label. My son is completely healthy, hasnt been sick since he started in Nov 2009, fev1 hovering around 89% (he's 32 and been in the trial). He uses pulmozyme sometimes...he isnt using Creon...
<br />
<br />This question above is asking whether the vx770 will work the same way for other mutations such as the RH117...hope that helps Gunelle <img src="i/expressions/face-icon-small-smile.gif" border="0"> It's VERY VERY exciting, hoping to get to the market by the end of this year, and they're working on another for df508 which is just starting initial ph 2 (the vx809) <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[Kristen]

I wanted to bump thread this with the news I shared in the other thread - they are going to test it on our mutation!
From this article:<a href="http://www.marketwatch.com/story/vertex-announces-key-2012-business-objectives-as-company-prepares-for-planned-global-launch-of-kalydeco-in-cystic-fibrosis-2012-01-08">http://www.marketwatch.com/story/vertex-announces-key-2012-business-objectives-as-company-prepares-for-planned-global-launch-of-kalydeco-in-cystic-fibrosis-2012-01-08</a>
<p id="">"As Vertex prepares for the potential launch of KALYDECO for people with the G551D mutation, the company is also planning to begin additional studies of KALYDECO in children with CF as young as two years of age and in people with CF who have certain mutations that were not evaluated in the previous Phase 3 studies. Pending final feedback from regulatory agencies, the company plans to begin three clinical studies of KALYDECO in mid-2012:
<p id="">-- Study in people with the R117H mutation: Vertex plans to begin the first clinical study of KALYDECO in people who have at least one copy of the R117H mutation in the CF gene. The R117H mutation causes abnormal function of the CFTR protein at the cell surface. Approximately 3 percent of people with CF in the U.S. have the R117H mutation."

 

[Kristen]

I wanted to bump thread this with the news I shared in the other thread - they are going to test it on our mutation!
From this article:<a href="http://www.marketwatch.com/story/vertex-announces-key-2012-business-objectives-as-company-prepares-for-planned-global-launch-of-kalydeco-in-cystic-fibrosis-2012-01-08">http://www.marketwatch.com/story/vertex-announces-key-2012-business-objectives-as-company-prepares-for-planned-global-launch-of-kalydeco-in-cystic-fibrosis-2012-01-08</a>
<p id="">"As Vertex prepares for the potential launch of KALYDECO for people with the G551D mutation, the company is also planning to begin additional studies of KALYDECO in children with CF as young as two years of age and in people with CF who have certain mutations that were not evaluated in the previous Phase 3 studies. Pending final feedback from regulatory agencies, the company plans to begin three clinical studies of KALYDECO in mid-2012:
<p id="">-- Study in people with the R117H mutation: Vertex plans to begin the first clinical study of KALYDECO in people who have at least one copy of the R117H mutation in the CF gene. The R117H mutation causes abnormal function of the CFTR protein at the cell surface. Approximately 3 percent of people with CF in the U.S. have the R117H mutation."

 

[missT]

super, duper excited about this news! Got it last week from my clinic but on line today! I am so hopeful.

 

[missT]

super, duper excited about this news! Got it last week from my clinic but on line today! I am so hopeful.

 

[marcijo]

I saw this study too-I am so excited about it! I have an email into my clinic and I have also talked to my old clinic in Seattle about it. I am going to keep on it and make sure I am a part of the study!

Exciting news!!!

 

[marcijo]

I saw this study too-I am so excited about it! I have an email into my clinic and I have also talked to my old clinic in Seattle about it. I am going to keep on it and make sure I am a part of the study!

Exciting news!!!

 

[Kristen]

That is great! I'd love to hear if any of you get into the trial. I'd love to, but can't because my husband and I are planning to try for another baby soon.

 

[Kristen]

That is great! I'd love to hear if any of you get into the trial. I'd love to, but can't because my husband and I are planning to try for another baby soon.

 

[CysticlyFIT]

Does any one know what class 1717-1G>A is in? Is it the 'G' class as well? I have that mutation with 508Ddelta, But I can never find any information on and it is never mentioned anywhere at CF conferences, CF research or Vertex. All my Doctor told me was one time " it may work a little bit, you're mutation'