A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...

"Monumental" is how Ashley E. Webb, MD Assistant Professor, Department of Pediatrics, Division of Child & Adolescent Neurology Neuromuscular Program Director, UTHealth, University of Texas Health ...

Fast functional testing of genetic variants, from newborn genomes to disease models like zebrafish, is transforming ambiguous DNA findings into confident, real‑time treatment decisions.

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

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 ...

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 ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...

Discover how a 10-year-old boy with spinal muscular atrophy finds freedom and strength through swimming, inspiring others to defy physical limits. Read more at straitstimes.com. Read more at ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. So most recently, there has been the ...

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 ...

I feel like my whole life has done a 360,” says Jesy Nelson in the final five minutes of Jesy Nelson: Life After Little Mix (Prime Video), the six-part documentary charting her life since leaving ...