PTC Initiates Phase 2 Clinical Trial of Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy in Pediatric Patients Between the Ages of Two and Five Years

PTC Therapeutics has announced that they have initiated a Phase 2 clinical trial of Translarna™ (ataluren) for children 2 to 5 years old. Details of site locations, etc. are on  Below is the announcement from PTC.

We are pleased to inform you that PTC Therapeutics has initiated a Phase 2 clinical trial of Translarna™ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD), in patients between the ages of two and five (they cannot have turned five to participate). This open-label, multiple-dose study will evaluate the safety and pharmacokinetics (PK) of Translarna in pediatric patients.

The study includes a 4-week screening period, a 4-week study period and a 48-week extension period for patients who complete the 4-week study period, for a total of 52 weeks of treatment.

For more information about the study and a list of participating study centers, please visit  

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