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 »
Our industry partners at Genentech recently released an SMA community letter summarizing 2022 developments and initiates. Check it out here!
Genentech Releases SMA Community Letter Read More »
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 Federal Aviation Administration (FAA) reauthorization bill. The last time the law was updated (2018), Congress included several provisions aimed at making air travel more accessible for people with disabilities. (For
Share Your Air Travel Concerns & Experiences for Cure SMA Report to Congress Read More »
