Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease. This is a ...
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 ...
The newly licensed intrathecal gene therapy expands life-changing treatment options for children and adults affected by ...
NBS programs are fairly new in the U.S., so not much is known about variations among them or about provider practice patterns for newborns with SMA. In a survey of providers and state NBS programs, ...
Novartis’ ITVISMA, approved a month ago by the US FDA, used at Sheikh Khalifa Medical City under the supervision of the ...
New York, Feb. 12, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant approval of a risdiplam ...
Please provide your email address to receive an email when new articles are posted on . The study examined 26 newborns with spinal muscular atrophy given Evrysdi. Data showed 81% of babies were able ...
The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...
Scholar Rock's stock soars more than 315% on positive trial of treatment for spinal muscular atrophy
Scholar Rock Holding Corp.'s stock soared more than 315% Monday to put it on track for its biggest ever one-day gain, after the clinical-stage biotech company said a late-stage trial of a treatment ...
ESTACADA, Ore. — A child from Estacada is progressing well after a new treatment to help with her spinal muscular atrophy. All was going well when Lily Ahrens was born, said her mom, Alysa Ahrens.
3don MSN
Boy, 5, Born with Rare Genetic Disorder Walks by Himself After Receiving World’s Most Expensive Drug
Megan Willis' son, Edward, was given the gene therapy, Zolgensma, after being diagnosed with spinal muscular atrophy when he ...
DUBLIN--(BUSINESS WIRE)--The "Spinal Muscular Atrophy (SMA) Treatment Market Size, Share & Trends Analysis Report By Route of Administration (Oral, Intrathecal), By Treatment (Gene Therapy, Drug), By ...
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