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G542X mutation-PTC 124
- Ερώτηση
- I ‘ve just learned about the clinical development stop for Ataluren. My son has G542X mutation, I would like to know to if there are other ongoing clinical trials?
Thank you
- Απάντηση
- Hello
Regarding experimental treatment to restore a functional CFTR protein (like Ataluren for nonsense mutations) there is no ongoing clinical trial for patients carrying a nonsense mutation (like G542X ).
Researchers work with multiples approaches to find a way to restore a functional CFTR protein for all classes of mutations. Those research is at a fundamental research step or at pre-clinical steps.
If you want to know trials you can take part in please contact your CF cares center to get information about the research protocols in progress in the center.
Best regards
Anna Ronayette
- 26.05.2017
