• Latest Posts

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

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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

Milestone for gene-edited therapy for sickle cell disease

Selecta and Astellas sign LOPD treatment agreement 

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