PTC Therapeutics Announces Positive Data from its Translarna™ Phase II Clinical Trial in Children as Young as Two Years with Nonsense Mutation Duchenne Muscular Dystrophy | DuchenneXchange

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PTC Therapeutics Announces Positive Data from its Translarna™ Phase II Clinical Trial in Children as Young as Two Years with Nonsense Mutation Duchenne Muscular Dystrophy


PTC Therapeutics, Inc. today announced the presentation of data from the Translarna (ataluren) Phase II Study 030 demonstrating that the safety and pharmacokinetic profile of Translarna in children from two to five years with nonsense mutation Duchenne muscular dystrophy (nmDMD) was consistent with that for older children.1 Importantly, the data also showed that treatment with Translarna resulted in improvements in timed function tests and the North Star Ambulatory Assessment from baseline at weeks 28 and 52, with mean changes showing as much as a 25 percent improvement after one year.

The data at 28 weeks formed the basis of the recent positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to expand the current indication of Translarna to include nmDMD ambulatory children from two to five years of age.2 The data was presented at the International Congress on Neuromuscular Diseases in Vienna.

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