Search Results: spinraza
The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants
Today, Lancet published results from the Phase 2, open-label trial of nusinersen (Spinraza) in infants. This trial initiated in 2013, and patients continue to receive […]
Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study
Biogen and Ionis Pharmaceuticals announced that SPINRAZA (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of […]
Cure SMA Announces the Publication of “Impact of Early Intervention on Motor Milestone Achievement in Spinal Muscular Atrophy”
Cure SMA is pleased to announce the publication of “Impact of early intervention on motor milestone achievement in spinal muscular atrophy: Insights from Cure SMA […]
2026 Annual SMA Research & Clinical Care Meeting: Session Summaries
Make today a breakthrough. At the end of June, SMA researchers and clinicians from around the world – spanning academia, government, and biotech and pharmaceutical […]
Meet the Scientists Behind SMA Research and Discover What’s Coming Next
The Family-Friendly Poster Session is one of the most anticipated events at the Annual SMA Conference! This year’s session will be held on Friday, June […]
Cure SMA Awards $150,000 Grant to Emma Sutton, PhD, at Ottawa Hospital Research Institute in Canada
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal […]
SMA Treatment Delay FAQ
Make today a breakthrough. Last Updated: 09/11/26 We know our community has many pressing questions related to the FDA approval delay of Scholar Rock’s […]
Cure SMA Update on SMA Treatment Delays
Dear SMA Community, Yesterday was a disappointing and frustrating day for our community. We learned of two FDA approval delays for Scholar Rock’s apitegromab and […]
Biogen Receives News on Supplemental New Drug Application (sNDA) for High Dose Nusinersen from the U.S. FDA
Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the supplemental New Drug […]
Biogen Announces Advancement of Investigational SMA Drug Salanersen to Registrational Trials Based on Positive Phase 1 Results
“To see a child dosed with gene therapy at age one and still unable to sit at age five then gain that ability within three […]