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clinical trial
- Question
- Hello
Is there any clinical trial regarding patients with mutation G542X/Delta F508 ?
Thank you - Answer
- Hello
Today, there is no clinical trials specific for patient with the mutations G542X/Delta F508.
However, a trial regarding patient with at least one non-sense mutation (like G542X, class I) will start soon in France to assess the efficacy of a molecule named Ataluren (https://clinicaltrials.gov/ct2/show/NCT02139306?term=ACT+CF&rank=1). Ataluren could correct the mutation and lead to the generation of a normal CFTR protein.
Other studies, planned by Vertex Company, for patients heterozygous for the mutation F508del, are coming.
Best regards,
Anna Ronayette
Head of clinical research programs of Vaincre la mucoviscidose
- 13.03.2015
