INDIANAPOLIS — An Indiana man took a trip to Europe thanks to his friends and hundreds of generous strangers. Kevan Chandler, 30, suffers from spinal muscular atrophy, which has left him confined to a ...

If you’ve wondered if spinal muscular atrophy is the same as spina bifida, you aren’t alone. It’s easy to confuse these two conditions since they both involve the spine. But spinal muscular atrophy ...

Spinal muscular atrophy and muscular dystrophy both affect muscle size and function, causing weakness and leading to health complications like difficulty walking, swallowing, and, for some people, ...

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 ...

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 ...

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 ...

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 ...

For patients with nonambulatory type 2 or type 3 spinal muscular atrophy, apitegromab, a monoclonal antibody that inhibits myostatin activation, improves motor function versus placebo, according to a ...

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