England will add SMA testing to its newborn screening program from October 2026, expanding nationwide by October 2027 to help ...

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 ...

Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

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 ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...

This image from video provided by UPMC and University of Pittsburgh Health Sciences shows Doug McCullough, who has spinal muscular atrophy, during tests of experimental spinal cord stimulation to ...

The teen's mother said he first began showing symptoms at 2 months old when “his arms and legs were just hanging by his side” ...

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...