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FDA grants breakthrough therapy designation to Duchenne muscular dystrophy treatment

Boost for Charcot-Marie-Tooth patients as Novartis acquires DTx Pharma


Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy

Fabry disease treatment gets EU marketing authorization

Orphalan announces U.S. launch of drug to treat Wilson disease

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

AI boosts genome editing