Latest News
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, for the treatment of spinal […]
Read More ›District of Columbia Starts Screening for SMA During Rare Disease Month
Cure SMA is pleased to announce that the District of Columbia (DC) is now screening babies born in DC for spinal muscular atrophy (SMA). The DC Department of Health (DC […]
Read More ›NEW REPORT: SMA Community Describes Air Travel Experiences as “Good, but mostly Bad, and Ugly”
Despite federal laws and protections aimed at improving air travel for people with disabilities, a new report from Cure SMA highlights that the air travel experience for most individuals and […]
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 Atrophy (SMA).” Dr. Rage’s basic […]
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. Blatnik’s basic research grant is […]
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. In addition, many more potential […]
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