"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 ...
—A new qualitative participatory study explored how powered mobility (PM), in the form of modified ride-on cars, impacts children with spinal muscular atrophy type 1 (SMA1) and their families. Here ...
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WASHINGTON, Feb. 3, 2026 /PRNewswire/ -- Cure SMA today announced that key research and legislative priorities championed by the spinal muscular atrophy (SMA) community have been approved by Congress ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...
In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene ...
A single dose of a new formulation of the gene replacement therapy intrathecal onasemnogene abeparvovec ( OAV101 IT) improved movement ability in teens and children older than 2 years with spinal ...
Waking up, hopping out of the bed, and stumbling to the kitchen for a cup of coffee: It’s an everyday routine most people don’t think twice about. But for children with spinal muscular atrophy, simply ...
Health Secretary Wes Streeting questioned on lack of newborn test for spinal muscular atrophy (SMA) to stop more parents from ...
Kerala government allocates ₹30 crore in budget for Spinal Muscular Atrophy treatment, supporting families of affected children.
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