Research
Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh
Lyndsay Murray, PhD, was awarded $75,000 for her research project, “Evaluating the impact of sequential versus simultaneous administration of SMN-inducing compounds on motor unit recovery […]
Read More ›Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy
This week, Biohaven announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, […]
Read More ›Cure SMA Awards $150,000 Grant to Florence Rage, PhD, at the Institute of Molecular Genetics of Montpellier
Florence Rage, PhD, was awarded $150,000 for her research project, “Deciphering the Mechanisms of RNA Transport along Axons to Understand the Progression of Spinal Muscular […]
Read More ›Cure SMA Awards $150,000 Grant to Anton Blatnik, PhD, at The Ohio State University
Anton Blatnik, PhD, at The Ohio State University has been awarded $150,000 for his research project, “Determining Primary Splicing Changes in Spinal Muscular Atrophy.” Dr. […]
Read More ›Cure SMA Supports Clinical Trial Readiness
In recent years, the FDA-approval of three new SMA treatments has made it possible for a growing number of affected individuals to receive disease-modifying drugs. […]
Read More ›Cure SMA Awards $150,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires
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 Holds Patient-Led Listening Session with FDA
On Thursday, August 4, 2022, six members of the SMA community had the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) […]
Read More ›Cure SMA Holds Patient-Led Listening Session with FDA
On Thursday, August 4, 2022, six members of the SMA community had the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) […]
Read More ›Cure SMA Launches Benefit-Risk Survey to Assess Patient Perspective on Treatment Possibilities and Risks
Cure SMA is conducting a survey to understand our community’s perspectives on treatment possibilities and tolerance for the side effects of currently available and planned […]
Read More ›2022 Cure SMA Annual Research Meeting Summaries – Special and Clinical Research Session
In June, SMA researchers and clinicians from around the world met in Anaheim, CA, for the 2022 Annual SMA Research and Clinical Care Meeting. There […]
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