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

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

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

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

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

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

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

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, ...

spinal muscular atrophy, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the ...