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 muscular atrophy (SMA). Fast Track designation enables important new drugs to reach patients earlier by facilitating more frequent communications with the FDA and expeditious review […]
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 Health) confirmed its screening news with Cure SMA. “DC Health is pleased to announce that as of February 1, babies born in the District of
District of Columbia Starts Screening for SMA During Rare Disease Month Read More »
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 families with spinal muscular atrophy (SMA), a neuromuscular disease, remains “mostly bad and ugly”. Individuals and families with SMA, a disease that significantly impacts physical
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 research grant is one of four awarded by Cure SMA in 2022 totaling $500,000. Meet Dr. Rage Tell us about yourself. I am a neuroscientist
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 one of four awarded by Cure SMA in 2022 totaling $500,000. Meet Dr. Blatnik Tell us about yourself. I am a lover of music, hard
Cure SMA Awards $150,000 Grant to Anton Blatnik, PhD, at The Ohio State University Read More »
