Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

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 ...

Spinal and bulbar muscular atrophy (SBMA) is a rare X-linked neuromuscular disorder that results from an abnormal expansion of gene sequences in the androgen receptor gene. SBMA has an adult onset and ...

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterised by the loss of alpha motor neurons in the anterior horn of the spinal cord, leading to progressive muscle ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

The singer has renewed her campaign for universal newborn screening for spinal muscular atrophy as MPs prepare to debate the issue in Parliament following a petition backed by more than 150,000 ...

Former Little Mix singer Jesy Nelson has described the roll-out of spinal muscular atrophy (SMA) screening as a “postcode ...

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study ...

Biogen has won U.S. Food and Drug Administration breakthrough-therapy designation for its proposed salanersen treatment for the neuromuscular disease spinal muscular atrophy, or SMA. The Cambridge, ...