We’re happy to share our next recipient of Cure SMA’s basic research funding, Allison Ebert, PhD, at The Medical College of Wisconsin!   Read more about her project in our news section: curesma.org/ebert-grant-an…

Scholar Rock recently shared new data showing treatment with apitegromab was linked to improved muscle strength and range of motion, while being safe and well-tolerated in those with SMA types 2 and 3.   To learn more, visit out news section: curesma.org/scholar-rock-n…

This weekend, the President signed into law a government funding bill that included a provision in support of new SMA research at the National Institutes of Health (NIH).   Visit our news section to learn more: curesma.org/gov-funding-bi…

We’re happy to share our next recipient of Cure SMA’s basic research funding, Michael Tellier, PhD, at the University of Leicester! Dr. Tellier was awarded $114,000 for his research project, “Characterization of the SMN-7SK complex in the regulation of SMN1 and SMN2 expression

Scholar Rock is proud to be a Title Sponsor of the Cure SMA 2024 Annual SMA Conference and the Annual SMA Research & Clinical Care Meeting. We look forward to seeing the #SMA community this week in Austin! #2024SMAConference

Join streamer Simhamil live on Twitch NOW! At conference, stop by the Cure SMA Hub in Griffin Hall while you eat lunch. At home, chat with other community members virtually while Sim plays the sims! twitch.tv/sim_simmy

Streamers Simhamil and @athenahighland will be live on Twitch this afternoon for the Cure SMA annual conference! Come chat with other streamers, gaming enthusiasts, conference attendees, and SMA community members at 11:30am and 1:15pm CT. We'll see you online!

Today, Scholar Rock announced that its SAPPHIRE Phase 3 Study achieved its primary endpoint demonstrating a statistically significant and clinically meaningful improvement for apitegromab. The company plans to submit a U.S. Biologics License Application (BLA) and a European Union

Biogen today announced that data from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals previously treated and treatment-naïve to nusinersen.   Learn more: curesma.org/new-higher-dos…

Discover the latest advancements in Spinal Muscular Atrophy (SMA) diagnosis with leading experts Kapil Arya, MD, Juan Francisco Vazquez Costa, MD, PhD, and Mary Schroth, MD. In this in-depth 6-part series, these neurologists delve into new diagnostic guidelines, treatment access

Last week, Genentech presented positive 2-year data from the ongoing RAINBOWFISH study. The data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before 6 weeks of age, with most achieving motor milestones similar to children without SMA. All

Don't miss the final episode in our 6-part panel series discussing the lates updates in spinal muscular atrophy (SMA) diagnosis. Kapil Arya, MD, Juan Francisco Vazquez Costa, MD, PhD, and Mary Schroth, MD in a presentation for the medical community. Please share this with your

As part of our ongoing efforts to drive quality health care standardization and improvements for the SMA community, Cure SMA is pleased to announce the next publication in a series of resources designed to support healthcare professionals, and individuals with SMA, and their

Are you a healthcare professional looking for educational opportunities? Check out the educational opportunities available for clinicians here! lnkd.in/gWHbYntE For an opportunity to be awarded one free registration and 3 nights hotel to the 2025 Annual SMA Research &