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Research Update

Question: Hello,

Could we have a French synopsis on positive results regarding research (Vertex, QR-010, PTC124, others?) for patient heterozygous for the F508del mutation?
Thank you.
Answer: Hello,

Several clinical trials for patients heterozygous for the F508del mutation are ongoing or just ended. These trials evaluate the effects of various molecules as VX661 (Vertex), Roscovitine (Manros-therapeutics), GS-5745 (Gilead), and results are not yet available.
Clinical trials to evaluate drugs like QR-10 (ProQR) and JBT-101 (Corbus) should begin before the end of the year.
The increase of clinical trials intended for patients heterozygous for the F508del mutation are the proof of positive results regarding the development steps of new therapies that allow to provide patients with cystic fibrosis effective treatments.

Best regards
Anna Ronayette; 19.09.2016

ECORN-CF European Centers of Reference Network for Cystic Fibrosis

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