Biogen has secured European regulatory approval for a high-dose regimen of its Spinraza drug for the neuromuscular disease spinal muscular atrophy, or SMA. Biogen on Monday said the European ...

Amsterdam, February 14, 2023 – A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...

Children with spinal muscular atrophy (SMA), a rare autosomal recessive disease that results in progressive muscle weakness and atrophy, can suffer the concerning consequences of dysphagia, which ...

Taldefgrobep alfa, in Phase 3 global clinical development for Spinal Muscular Atrophy, granted EU Orphan Drug Designation in addition to previously receiving Fast Track and Orphan Drug Designation in ...

Jesy Nelson of Little Mix fame has shared the news that her twin girls have been diagnosed with SMA Type 1, a genetic ...

On Sunday, Jesy Nelson, a former member of beloved girl group Little Mix, shared the emotional news that her eight-month-old ...

The Little Mix singer, 34, revealed her eight-month-old daughters suffer from a severe muscular disease and will likely be ...

Scholar Rock Holding Corp.'s stock soared more than 315% Monday to put it on track for its biggest ever one-day gain, after the clinical-stage biotech company said a late-stage trial of a treatment ...

The former Little Mix singer posted an emotional video to Instagram on Sunday, vowing that the girls will "fight all the odds ...