A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Data from the SAPPHIRE study formed the basis of Scholar Rock's U.S. Food and Drug Administration request to approve ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...

Preclinical data with the investigational gene therapy INS1202 show improvements in survival and motor performance in ALS.

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ...

Scholar Rock will present new Phase 3 SAPPHIRE trial data on apitegromab at the 2025 MDA conference, highlighting efficacy ...

Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...

Roche (RHHBY) announced that it will present new data at the Muscular Dystrophy Association, MDA conference, 16-19 March, 2025, in Dallas, ...

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...