Research
Cure SMA Awards $50,000 Grant to Tianyuan Shi, PhD, at the University of Hong Kong
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA […]
Read More ›Cure SMA Awards $150,000 Grant to Charlotte Sumner, MD, at Johns Hopkins University School of Medicine
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA […]
Read More ›Check Out Cure SMA’s 2024 Annual State of SMA Report
Check Out Cure SMA’s 2024 Annual State of SMA Report Cure SMA is pleased to announce the launch of our next State of SMA report, […]
Read More ›SMA Community Risk Tolerance Update: Comparison of 2022 and 2017 SMA Risk/Benefit Survey Data
One of Cure SMA’s top priorities is to relay the SMA community’s treatment experiences and preferences to the United States Food and Drug Administration (FDA). […]
Read More ›Novartis Intrathecal Gene Therapy Onasemnogene Abeparvovec Phase III Study Meets Primary Endpoint in Children and Young Adults with SMA
Novartis today announced positive topline results from the Phase III STEER study. This pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec […]
Read More ›Biohaven Provides Update on Taldefgrobep Alfa Development Program for Spinal Muscular Atrophy
In the RESILIENT SMA study, Biohaven announced today that taldefgrobep alpha showed clinically meaningful improvements in motor function at all time points on the Motor […]
Read More ›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 […]
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