Treatment with salanersen slowed neurodegeneration and improved motor function in children with SMA in a Phase 1 trial, ...

New data from Biogen reveals higher Spinraza doses may enhance motor function in SMA patients. Read more here.

Biogen plans to advance an experimental treatment for the neuromuscular disease spinal muscular atrophy, or SMA, into registrational studies following encouraging results from an early-stage trial.

The cumulative data from the Phase 1 study indicate that salanersen has a generally well tolerated safety profile at both the 40 mg and 80 mg doses, with most adverse events (AEs) mild to moderate in ...

Many SMA patients continue to see gains in motor function with long-term Spinraza treatment, a real-world study from ...

She dreams that she’s not in a wheelchair. She dreams that she can run." The post Girl diagnosed with rare muscle condition ...

For the first time in India, a newborn diagnosed with Spinal Muscular Atrophy (SMA) received pre-symptomatic treatment within days of birth in Kerala, marking a significant milestone in the treatment ...

DataM Intelligence | competitive Intelligence Spinal Muscular Atrophy treatment is evolving fast, with gene therapy, oral drugs, and next-g ...

5q-Spinal muscular atrophy (SMA) is a progressive, autosomal recessive neuromuscular disease with an incidence of 1 in 14,848 births.1 SMA is characterised by proximal muscle weakness and in severe ...

June 17th 2019 EP. 7: Spinal Muscular Atrophy (SMA) and Types of SMA June 17th 2019 EP. 8: Patient Perspective and Payer Perspective of SMA June 17th 2019 EP. 9: Payer Diagnostic Criteria and ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene ...