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

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 ...

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

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

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

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

Bay County officials say the beach access construction is almost complete. Bay County has 27 miles of sandy beaches and ...

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

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

The study was originally presented at the 2024 MDA Clinical & Scientific Conference, view the abstract here. About Spinal Muscular Atrophy (SMA) SMA is caused by a mutated or missing survival ...