vx770 for some other mutations!

[beleache]

Wonderful news !! While I dont have any of these mutations I am thrilled that so many mutations are added to the list !! I hope & pray that eventually all mutations will be covered !! <br>Happy day all <img src="i/expressions/face-icon-small-smile.gif" border="0"> joni

 

[beleache]

Wonderful news !! While I dont have any of these mutations I am thrilled that so many mutations are added to the list !! I hope & pray that eventually all mutations will be covered !! <br>Happy day all <img src="i/expressions/face-icon-small-smile.gif" border="0"> joni

 

[samaruba]

mama Scarlett,
I know how you feel.... shocked and very confused. I just heard it 5 days ago..... My head is still spinning....
I too was happy for all with G551D and hoping they'd be close to finding something that would work for my son and ...boom.... I get an email 5 days ago with this news.... I really never expected this! I just didn't know what to do, what to think and what to feel...... I still don't really know how I feel..... it's just too weird.... but I couldn't be happier!
Hoping that they'll move fast on developing something for everyone!
Sam

 

[samaruba]

mama Scarlett,
I know how you feel.... shocked and very confused. I just heard it 5 days ago..... My head is still spinning....
I too was happy for all with G551D and hoping they'd be close to finding something that would work for my son and ...boom.... I get an email 5 days ago with this news.... I really never expected this! I just didn't know what to do, what to think and what to feel...... I still don't really know how I feel..... it's just too weird.... but I couldn't be happier!
Hoping that they'll move fast on developing something for everyone!
Sam

 

[KMV77]

Just got a call today about a program where I can start taking the vx770 now. Going for bloodwork, doing paperwork, and I should be on it in too weeks. Cant wait to see what it does to these old lungs.
Kevin

 

[KMV77]

Just got a call today about a program where I can start taking the vx770 now. Going for bloodwork, doing paperwork, and I should be on it in too weeks. Cant wait to see what it does to these old lungs.
Kevin

 

[Kristen]

Whooohooo! I just read this today: 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: -- Pediatric study: A study of KALYDECO in children ages 2 through 5 with gating mutations, including G551D, is expected to evaluate the safety, tolerability and effect on sweat chloride and other measures of clinical activity using a pediatric formulation of KALYDECO. -- 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. -- Study in other gating mutations: Vertex also plans to begin the first clinical study of KALYDECO in other gating mutations where CFTR proteins are present at the cell surface but do not function properly. G551D is the most common gating mutation, present in approximately 4 percent of people with CF in the U.S., and was the focus of previous Phase 3 KALYDECO studies. The remaining gating mutations to be evaluated in this study account for an additional approximately 1 percent of people with CF in the U.S. From this link: <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>

 

[Kristen]

Whooohooo! I just read this today: 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: -- Pediatric study: A study of KALYDECO in children ages 2 through 5 with gating mutations, including G551D, is expected to evaluate the safety, tolerability and effect on sweat chloride and other measures of clinical activity using a pediatric formulation of KALYDECO. -- 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. -- Study in other gating mutations: Vertex also plans to begin the first clinical study of KALYDECO in other gating mutations where CFTR proteins are present at the cell surface but do not function properly. G551D is the most common gating mutation, present in approximately 4 percent of people with CF in the U.S., and was the focus of previous Phase 3 KALYDECO studies. The remaining gating mutations to be evaluated in this study account for an additional approximately 1 percent of people with CF in the U.S. From this link: <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>

 

[marcijo]

I saw that article too Kristen-I am SUPER excited about it and am going to be a part of the R117H study no matter what! <img src="i/expressions/face-icon-small-smile.gif" border="0">
I don't think my clinic will be doing the study-but there is a clinic 6 hours away (my old clinic) that I am hoping will particpate.
Great news!

 

[marcijo]

I saw that article too Kristen-I am SUPER excited about it and am going to be a part of the R117H study no matter what! <img src="i/expressions/face-icon-small-smile.gif" border="0">
I don't think my clinic will be doing the study-but there is a clinic 6 hours away (my old clinic) that I am hoping will particpate.
Great news!