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 in mouse models of SMA.” Dr. Murray’s basic ...
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). ...
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 ...
Saravanan Arumugam, PhD, was awarded $100,000 for his research project, “Role of Cytosolic and Mitochondrial Ca2+ in Homeostasis the Pathogenesis of Spinal Muscular Atrophy.” Dr. Arumugam's basic research grant is one of five ...
Anton Blatnik, PhD, at The Ohio State University has been awarded $100,000 for his research project, “Determining Primary Splicing Changes in Spinal Muscular Atrophy.” Dr. Blatnik's basic research grant is one of five ...
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 SMA treatments are ...
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 biology. Our Scientific Advisory Board ranks the submitted ...
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) about their lived experiences with SMA and their ...
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 therapies. The results will help inform our future ...
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 they shared their most recent data with the ...
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 they shared their most recent data with the ...
Congress has recognized the unmet and new needs of individuals with SMA by including legislative language in must-pass legislation asking the National Institutes of Health (NIH) to continue research in SMA. At the ...
