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    Kalydeco New Drug Application submited to FDA for R117H for 18 yo and older

    It is official the application has been submitted to the FDA for approval. I was told this process takes 6-9 months. Wish this was done 6-9 months ago when the test was completed, but they had to rethink the application since the test endpoints were poorly defined. I am very thankful that...
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    Addition of VX-661 to KALYDECO(R) (ivacaftor) Improves Lung Function short study P2

    At first, I was a little confused about the results from this 4 week study. Here is my summary of what happened. They took people who were already taking Kalydeco and then also gave some of them VX-661. These people had one gene that was DelF508 and the other gene was one of the G551D...
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    Kalydeco gets approval for eight more mutations

    Good news for approx. 150 people with CF 6 and older. a supplemental New Drug Application (sNDA) for KALYDECOTM (ivacaftor) for people with cystic fibrosis (CF) ages 6 and older who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene...
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    Vertex Ivacaftor R117H Study Results -Fixes everything that CF breaks for this group

    Is it just me or was this study poorly defined, providing misleading results? I understand this was a difficult study to perform with so few participants, but it should have been tailored to the population. They took patients under 12 who had FEV1 between 40 - 105% and patients above that...
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