"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 ...

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 ...

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 ...

The Government is required to respond to the petition if it reaches 10,000 signatures.

"The strongest, most resilient babies," the Little Mix singer said of her daughters Ocean Jade and Story Monroe ...

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 ...

Jesy Nelson is continuing to ramp up her campaigning for Spinal Muscular Atrophy (SMA) awareness while her music career takes a back seat. On 4 January, the singer shared the devastating news that ...

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 ...

A mum has been left "overwhelmed" after a fundraiser raised more than £5,000 to help her purchase a spinal brace for her son.

Jesy Nelson hasn’t given up hope that her twin daughters, Ocean Jade and Story Monroe, will overcome a muscle disease they were diagnosed with a month ago. The former Little Mix singer was recently a ...