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

(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)