Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...

In 2024, Pfizer reported a death that occurred in a phase II study of fordadistrogene movaparvovec, an investigational agent for Duchenne muscular dystrophy; the company has since discontinued the ...

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 ...

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

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

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...

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 ...

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 ...

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 ...