PTC Therapeutics, Inc. completes enrollment of landmark trial in Duchenne muscular dystrophy

PTC Therapeutics, Inc. today announced that it has completed enrollment of ACT DMD, the Phase 3 confirmatory trial of Translarna (ataluren) for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Top-line data from the trial is expected in the second half of 2015 and will support further approvals globally, following European approval received earlier this year.

ACT DMD is a 48-week clinical trial designed to confirm the effect of Translarna on ambulation in patients with nmDMD. The primary endpoint is the change in walking distance as measured by the six-minute walk test. The trial has reached full enrollment across 54 sites globally. All patients in the trial are eligible to participate in an open-label extension study, which has already begun enrolling patients who have completed the initial 48 weeks of treatment.

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