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US FDA approves PTC Therapeutics’ gene therapy for ultra-rare disorder

Summary by WTVB
(Reuters) – The U.S. Food and Drug Administration approved PTC Therapeutics’ gene therapy that is administered directly in the brain to treat a potential fatal enzyme deficiency disorder, the company said on Wednesday. (Reporting by Sriparna Roy, Leroy Leo and Christy Santhosh in Bengaluru; Editing by Maju Samuel)
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