Cystic Fibrosis Forum (EXP)

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  1. M

    Ataluren-the drug that "fixes" the CF gene

    Friday I went to the doctor, and he told me I was eliagible for the phase 3 study of ataluren, the drug that fixes the "nonsense" CF mutation. My mutation is R553X/F508del, meaning that the CFTR gene is there, its just cut short, so it doesnt work. Ataluren finishes this protein, basically...
  2. M

    Ataluren-the drug that "fixes" the CF gene

    Friday I went to the doctor, and he told me I was eliagible for the phase 3 study of ataluren, the drug that fixes the "nonsense" CF mutation. My mutation is R553X/F508del, meaning that the CFTR gene is there, its just cut short, so it doesnt work. Ataluren finishes this protein, basically...
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