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 ...

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

"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 ...

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

"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 ...

An international study has discovered that people living with the neurodegenerative condition Spinal Muscular Atrophy (SMA) are at higher risk of developing fatty liver disease, suggesting that SMA ...

(RTTNews) - Genentech, a member of the Roche Group (RHHBY), announced that the U.S. Food and Drug Administration has approved a New Drug Application or NDA for an Evrysdi (risdiplam) tablet for people ...

SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting those impacted by spinal muscular atrophy (SMA), welcomes the FDA approval of ...

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 ...