The former Little Mix star opened up about Ocean Jade and Story Monroe's spinal muscular atrophy diagnosis, but says she ...

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

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

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

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 ...

Little Mix alum Jesy Nelson is opening up about her 9-month-old twin daughters’ battle with a severe muscle disease. Nelson, 34, expressed hope that her baby girls, Ocean Jade and Story Monroe, would ...

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 ...

"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 ...

"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 ...

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 fundraiser to benefit Working on Walking, a nonprofit that raises funds and awareness for Spinal Muscular Atrophy, will be held at Napolitano's Lounge & Brooklyn Pizza, 375 ...