We’d like to give a warm welcome to Children’s Hospital Colorado as a newly added Cure SMA Care Center! Thank you to Loree, Ward and Lyza Weisman for your generous donation supporting your local SMA Care Center – the Children’s Hospital Colorado. We also thank the Weisman Family Foundation and the Ritter Family Foundation. The […]
Cure SMA Welcomes New Care Center Network Site! Read More »
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 research grant is one of five awarded by Cure SMA in 2022 totaling $525,000. Meet Dr. Murray Tell us about yourself. I
Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh Read More »
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 »
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 being studied in ongoing clinical trials, either alone or alongside approved treatments. This rapid increase in SMA research has made it more
Cure SMA Supports Clinical Trial Readiness Read More »
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 proposals on both their scientific merit and relevance to the Cure SMA research priorities. This year, Cure SMA’s top basic research priorities
Cure SMA Awards $150,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires Read More »
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 SMA community in a new advocacy 101 video series. In conjunction with the new 118th Congress, Cure SMA released a series of short, online videos that provide background on Congress
Cure SMA Unveils Advocacy 101 Video Series in Conjunction with 118th Congress Read More »
