muscular dystrophy
Sarepta Licenses Clinical, Preclinical siRNA Candidates From Arrowhead, Enters Discovery Deal
Sarepta will pay Arrowhead Pharmaceuticals $500 million upfront, make a $325 million equity investment, plus pay $250 million over five years, and more.
Regenxbio Treats First Patient in Pivotal Stage of Duchenne Muscular Dystrophy Gene Therapy Trial
The company expects the trial to support the submission of a biologics license application with the US Food and Drug Administration in 2026.
Sarepta Stops Duchenne Drug Development; Nets Higher-Than-Expected Elevidys Q3 Sales
The firm is discontinuing its SRP-5051 development program but recorded $181.0 million in Elevidys sales.
Atamyo Therapeutics Files IND to Begin Testing Limb-Girdle Muscular Dystrophy Gene Therapy in US
The clinical trial testing ATA-200 to treat a type of LGMD caused by mutations in the SGCG gene has already been cleared in Italy and France.
During a panel discussion at the National Society of Genetic Counselors meeting, genetic counselors discussed the risks and benefits of gene therapies.