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p.W1204X Mutation
- Question
- Hello,
I wanted to know if the mutation carried by my daughter, the p.W1204X could possibly benefit from the drug that would result from project ataluren?
- Answer
- Hello,
P.W1204X mutation is a mutation (type I) that causes a premature stop signal (stop codon) and ataluren is known to have an effect on patients with this type of mutation.
Your daughter could therefore possibly benefit from ataluren.
Best regards
Anna Ronayette (Head of clinical research programs of Vaincre la mucoviscidose)
- 15.05.2012