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

Monday, Novartis AG (NYSE:NVS) presented new data for Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA). Final data from the SMART Phase ...

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 ...

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 ...

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 ...

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 ...

The FDA approved a risdiplam tablet for SMA patients aged 2+ and over 44 lbs, matching the oral solution's efficacy and safety. Risdiplam, an SMN2 pre-mRNA splicing modifier, enhances SMN protein ...

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 ...

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 ...

The "SMAshing My Limits" campaign centers on a digital resource hub that includes information about living with and managing SMA, patient stories, community support resources and downloadable guides. ...