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

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

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

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

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

These data demonstrate the breadth of Roche’s neuromuscular portfolio across spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). “These longer-term results are encouraging for ...

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.

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...

The therapy, EPI-321, is intended to treat cases of facioscapulohumeral muscular dystrophy, a genetic neuromuscular disorder.