nonsense mutation Duchenne muscular dystrophy
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PTC Therapeutics to file new Phase III data for genetic muscle disorder drug in Canada
March 15, 2016Manufacturing and Production, Research and Development Canada, PTC Therapeutics, Translarna, nonsense mutation Duchenne muscular dystrophy, phase III
PTC Therapeutics (Nasdaq: PTCT) said it does not expect the Canadian regulators to review its trial drug to treat a …
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