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Thread: CFTR modulation for those with ONE copy of DDF508?

  1. #1
    sasha
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    CFTR modulation for those with ONE copy of DDF508?

    Does anyone have info on cftr modulation drugs for people that carry only one copy of the DDF508 mutation, and another of..something else? My other mutation is a nonsense mutation. Are any clinical trials going on for those of us in this boat? Or is there any research on Kalydeco combined with other drugs for the patients with one ddf508? Thanks!

  2. #2
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    vertex is running one arm of a combo of vx661 and kalydeco in a phase 3 trial for those with one copy of df508 and a second copy that isn't believedto benefit. I'm putting your nonsense. Italian in that group but if you specify it,I can see if there is anything indicating it is one that might benefit with kalydeco...if so another arm has folks with a second mutation that is gating or residual and a fourth arm is dd508. Vertex said depending on how the first part of the trial goes it might expand the arm for df508 for heterozygotes. Here's the press release. http://investors.vrtx.com/releasedet...leaseID=890626
    also this year vertex will have a new corrector in clinic that next year all going well would be added to 661 and kalydeco for the three to target heterozygotes. Let me know if this doesn't make sense. There are other pharmas now research these areas too, but they aren't as far along and I don't k oe those specifics.

  3. #3
    Quote Originally Posted by sasha View Post
    Does anyone have info on cftr modulation drugs for people that carry only one copy of the DDF508 mutation, and another of..something else? My other mutation is a nonsense mutation. Are any clinical trials going on for those of us in this boat? Or is there any research on Kalydeco combined with other drugs for the patients with one ddf508? Thanks!
    Sasha my kids are in the same boat as well. They have one copy df508 and a nonsense mutation which no one else has py849x. We are hopeful.

  4. #4
    Quote Originally Posted by threebabies View Post
    Sasha my kids are in the same boat as well. They have one copy df508 and a nonsense mutation which no one else has py849x. We are hopeful.
    Mine too. DF508 and Q493X. Are any of these drugs or drug combos currently in trials expected to help?

  5. #5
    Hi everyone! My two daughters have the classical mutation df508 and the nonsense mutation R1162X (the 'Italian mutation).
    Does anyone know if Vertex will be offering any drug combos for patients with these two mutations?

  6. #6
    We are also hoping for the best. Our mutations are W1206X and df508.

  7. #7
    ToriMom
    Guest
    We are too!! R1066C missense mutation and the DF508 combo. Very anxious here!!
    Michelle (mom to Victoria, age 14, gtube)

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