Front Page 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, […]
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 […]
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 […]
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 Unveils Advocacy 101 Video Series in Conjunction with 118th Congress
Individuals with SMA and their families can learn how to advocate with their Members of Congress more effectively on issues important to them and the […]
Read More ›Genentech Releases SMA Community Letter
Our industry partners at Genentech recently released an SMA community letter summarizing 2022 developments and initiates. Check it out here!
Read More ›Share Your Air Travel Concerns & Experiences for Cure SMA Report to Congress
The law that governs air travel in the United States is about to expire. Next year, Congress must consider air travel changes through a new […]
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