MedPage Today

Newborn Screening for Spinal Muscular Atrophy: Early Diagnosis and Treatment

Regina Trollmann, MD, of the Division of Pediatric Neurology, Department of Pediatrics, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany, and colleagues, did a retrospective ...
The American Journal of Managed Care

Shifting the Treatment Paradigm in Spinal Muscular Atrophy

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 ...
Science Daily

New target found for treatment of spinal muscular atrophy

Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...
MedPage Today

First-Ever Prenatal Treatment for Spinal Muscular Atrophy Shows Promise

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 ...
MarketWatch

Cure SMA Welcomes Broad FDA Approval of Itvisma(TM) for Spinal Muscular Atrophy, Reinforces Commitment to Patient Access and Support

The MarketWatch News Department was not involved in the creation of this content. SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting ...
Forbes

New Gene Therapy Possible For Infants With Spinal Muscular Atrophy

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...
Healio

Evrysdi linked to significantly improved outcomes in spinal muscular atrophy at 5 years

Please provide your email address to receive an email when new articles are posted on . At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation. Data ...
Healio

Babies with spinal muscular atrophy able to sit up after 1 year of Evrysdi treatment

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 ...
KOIN 6

Mom says at-home treatment for Spinal Muscular Atrophy boosted daughter’s care

PORTLAND, Ore. (KOIN) – August is Spinal Muscular Atrophy awareness month, and the disease’s first ever at-home treatment is having a major impact. SMA is a rare nerve disorder that affects motor ...
Morningstar

Scholar Rock Application for Spinal Muscular Atrophy Treatment Gets FDA Approval

Scholar Rock's biologics license application for apitegromab was accepted by the Food and Drug Administration. The late-stage biopharmaceutical company said the regulator will review the application ...
Morningstar

Cure SMA Welcomes Broad FDA Approval of Itvisma™ for Spinal Muscular Atrophy, Reinforces Commitment to Patient Access and Support

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 ...