Research
Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate
Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before 6 weeks of age, with most achieving motor milestones similar to children […]
Read More ›Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA)
Today, Scholar Rock announced plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in Q1 2025. Scholar […]
Read More ›Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Real World Evidence Collaboration Initiatives
Cure SMA is pleased to announce the involvement of scientific leadership, in the 4th Scientific International Congress on SMA (March 14 – 16, 2024), the […]
Read More ›Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships
Earlier this summer, at the Annual SMA Research and Clinical Care Meeting, Cure SMA highlighted six new basic research grants totalling $750,000. Cure SMA is […]
Read More ›Cure SMA Publishes Update on the Birth Prevalence of Spinal Muscular Atrophy (SMA)
Cure SMA is pleased to announce the publication of “Newborn Screening and Birth Prevalence for Spinal Muscular Atrophy in the US” in JAMA Pediatrics on […]
Read More ›Special Session Highlights from 2024 Annual SMA Research & Clinical Care Meeting
Each year, Cure SMA collaborates with our Scientific Advisory Board to plan a Special Session to be held during the Annual Research & Clinical Care […]
Read More ›Clinical Research and Drug Development Session Highlights from 2024 Annual SMA Research & Clinical Care Meeting
During the first week of June, SMA researchers and clinicians from around the world met in Austin, Texas, for the 28th Annual SMA Research & […]
Read More ›Spring 2024 Issue of Compass Now Available
The Spring 2024 issue of Compass is now available online. This year, we are proud to announce six new research grants, totaling $750,000, to […]
Read More ›SMA Researchers and Clinicians from Around the World Gather at the 28th Annual SMA Research & Clinical Care Meeting
During the first week of June, SMA researchers and clinicians from around the world met in Austin, Texas, for the 28th Annual SMA Research & […]
Read More ›Check Out Cure SMA’s 2023 State of SMA Report
Cure SMA is pleased to announce the launch of the third State of SMA report. The annual data report highlights data from Cure SMA’s three […]
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