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

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 ...

"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 ...

More than two years after the Government committed to adding SMA to newborn testing, families are still waiting ...

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 ...

Weeks after Jesy Nelson shared her daughters’ Spinal Muscular Atrophy Type 1 diagnosis, she shared why she and fiancé Zion Foster decided to break up.

"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 ...

The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

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 ...