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Thread: FDA approves Kalydeco for 5 residual function mutations

  1. #1
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    FDA approves Kalydeco for 5 residual function mutations


  2. #2
    Junior Member
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    Sep 2016
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    WOW! That's great news! They said previously that they only considered one of those 5 (which would have been strange) but now all 5 are approved. I hope they find a strategy to move forward with the rest of residual function splice mutations, there are so many different ones. It seems that they didn't use in vitro testing to approve these and I have not seen any published results of in vitro testing of potentiator efficacy on splicing mutations so I wonder if in vitro results are actually unreliable when it comes to splicing defects?

  3. #3
    Super Moderator
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    Sep 2016
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    Thanks for always keeping us informed of latest news Aboveallislove! This is wonderful news!
    45 y/o with CF (R334W and DF508)

  4. #4
    Junior Member
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    Sep 2017
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    Just got a call yesterday from my doctor that my mutation is one of them! SO happy!! Now I'm hoping my insurance will cover it *crosses fingers*.

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