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 ...

STELLAR-2, a randomized, double-blind, sham-controlled study, will evaluate the effects of salanersen when initiated ~6 months after onasemnogene abeparvovec-xioi in infants with SMA who received ...

A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies ...

Waking up, hopping out of the bed, and stumbling to the kitchen for a cup of coffee: It’s an everyday routine most people don’t think twice about. But for children with spinal muscular atrophy, simply ...

Spinal muscular atrophy (SMA) is a severe neurological disease caused by reduced survival of motor neuron (SMN) protein levels. There is presently no cure for the condition, although current therapies ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

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 ...

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 ...

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. Genetic factors are very important in ...