Ataluren (PTC124™) Clinical Trials

Nonsense Mutation Duchenne and Becker Muscular Dystrophy (nmDMD/BMD)

ACTIVE-NOT RECRUITING:
Phase 2b nmDMD/BMD Trial

PTC has fully enrolled a Phase 2b clinical study of ataluren in nmDMD/BMD. The trial is a multi-center, randomized, double-blind, placebo-controlled study to determine whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time. The trial is expected to conclude in 2010. More information on the trial can be found at www.clinicaltrials.gov (Search on "PTC124").

Nonsense Mutation Cystic Fibrosis (nmCF)

NOT YET RECRUITING:
Proposed Phase 3 CF Trial

PTC is planning to initiate a longer-term, Phase 3 clinical study of ataluren in patients with nonsense mutation CF within the next six months. The main goal of this study will be to understand whether ataluren can improve how nmCF patients feel and function and whether the drug can safely be given over a long period. The trial will be a multi-center, randomized, double-blind, placebo-controlled study. Additional details will be posted as they become available.

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