Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and latent forms of myostatin in order to inhibit myostatin activation.

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...

A cholesterol receptor protein called LDLR appears to be a key driver of neurotoxicity in sporadic ALS, per new research ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

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

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

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

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

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

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