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

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 ...

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

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 ...

Findings from DEVOTE support clinical benefits of the high-dose regimen of nusinersen (50 mg and 28 mg) in both ...

Biogen recorded declining sales for Spinraza in each of the four quarters of 2025, but the Massachusetts drugmaker sees ...

"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 ...

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 ...

"Managing pain in patients with SMA begins with the recognition that there are predictable etiologies leading to pain in SMA and investigating them during each medical visit is important to implement ...

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 ...