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PTC124

Question
I would like to know PTC124 results. Thank you
Answer
Hello,

The phase III clinical trial (Ataluren versus placebo) has ended on december 2011: 200 patients have been included, treated with Ataluren or placebo during one year.
The first results should be available at the summer 2012. Patient follow-up is ongoing, an open study which allow taking ataluren during 2 years, was offered to all participating patients in the phase III: clinicaltrials.gov/ct2/show?term=ataluren+cystic+fibrosis&rank=1.

If the study (ataluren versus placebo) shows an efficacy, a marketing authorisation will be asked by PTC firm. The delay for the commercialisation of the treatment is around 2 years.

Best regards
Anna Ronayette (Head of clinical research programs of Vaincre la mucoviscidose)
02.05.2012