Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease. This is a ...

Basel, July 2, 2026 – Novartis today announced that the European Commission (EC) has approved Itvisma ® (onasemnogene ...

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 ...

Ancient bacteria that have evolved to become integral to our cells—converting nutrients from food into energy—may also contribute to neurologic disorders, such as spinal muscular atrophy (SMA) and ...

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 ...

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 ...

Biogen is running a campaign aimed at spreading awareness of the next-gen version of its spinal muscular atrophy therapy ...

Regina Trollmann, MD, of the Division of Pediatric Neurology, Department of Pediatrics, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany, and colleagues, did a retrospective ...

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 ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in uterotreatment of SMA with ...