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 demonstrated by an analysis of data ...

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 ...

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 ...

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 ...

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 Inc. announced positive interim results from a Phase 1 study of salanersen (BIIB115/ION306), a novel antisense oligonucleotide for treating spinal muscular atrophy (SMA). The data show that ...

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

A retrospective analysis found glucocorticoid treatment to reduce comorbidities in adults with Duchenne muscular dystrophy (DMD) and assessed the relationship between anthropometric measures and ...

Spinal muscular atrophy is a rare genetic disorder causing progressive muscle weakness, now in focus after Jesy Nelson shared her twins' diagnosis.