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

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

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 ...

Zach Mecham has heard politicians demand that Medicaid recipients work or lose their benefits. He also has run into a jumble of Medicaid rules that effectively prevent many people with disabilities ...

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 ...

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.

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

Activating a protein called potassium channel Kv2.1 may help improve motor function in SMA, making it a potential SMA ...