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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

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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 

NMD Pharma cleared for spinal muscular atrophy trial

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