She slept in hospital intensive care units and navigated family outings with a breathing machine – all for her late son, who ...
More than two years after the Government committed to adding SMA to newborn testing, families are still waiting ...
The Generation Study, which is now available at Nottingham University Hospitals (NUH) NHS Trust, tests for more than 200 rare ...
The singer shared an emotional update about her twins’ health on Sunday.
"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 ...
New York, Jan. 26, 2026 (GLOBE NEWSWIRE) -- In recognition of Rare Disease Day on February 28, the Muscular Dystrophy Association (MDA) is sharing powerful community stories throughout the month that ...
Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...
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 ...
"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...
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 ...
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