Cure SMA

BREAKING: FDA approves Itvisma® (onasemnogene abeparvovec-brve) for treating spinal muscular atrophy (SMA) with a broad label covering all ages and types. This is a major step forward for the SMA community offering greater independence and new possibilities for those living with SMA.    Learn more: curesma.org/novartis-recei…   #SMA #TreatmentBreakthrough #CureSMA

Check out this story about our community member, Andrew Cherico 💜🧡

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 & Clinical Care Meeting, please submit your SMA education CME/CE certificates to patientcare@curesma.org.

This year’s Hope on the Hill event, which was held on November 19 in Washington, DC, recognized several lawmakers, including U.S. Representative Sam Graves of Missouri, U.S. Representative Pete Stauber of Minnesota, and U.S. Representative Mark DeSaulnier of California, U.S. Senator Tammy Baldwin of Wisconsin, and U.S. Senator Shelley Moore Capito of West Virginia.    During the event, several SMA community members highlighted Cure SMA’s year-long advocacy efforts to help address the needs of children and adults with SMA. Individuals with spinal muscular atrophy (SMA) and their families were also recognized as congressional champions.   Read more about the year’s event in our news section: curesma.org/sma-community-…

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 caregivers.       “Spinal Muscular Atrophy (SMA) Update in Best Practice: Recommendations for Treatment Considerations”, published in Neurology Clinical Practice, was developed in partnership with SMA key opinion leaders, individuals with SMA, and caregivers.    Learn more: curesma.org/update-in-best…

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 healthcare team. Episode 6: Monitoring Symptoms and Defining SMA Stages Stay informed on the ongoing work to refine SMA diagnosis and staging. This episode addresses how to monitor symptoms and define clinically meaningful stages for better care. neurologylive.com/roundtable-dis… To access the manuscript, click here: curesma.org/wp-content/upl…

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 children were able to swallow and feed orally, with none requiring permanent ventilation.   Learn more in our news section: curesma.org/genentech-evry…

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 strategies, and key insights from the latest research, providing crucial information for clinicians looking to improve patient outcomes. Episode 2: The Evidence Behind New Best Practices Dive into the clinical evidence and research that shaped the latest SMA diagnosis guidelines. Learn how decades of data have led to updated best practices. neurologylive.com/sma-diagnosis-… To access the manuscript, click here: curesma.org/wp-content/upl…

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…

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 marketing authorisation application (MAA) in Q1 2025.   Learn more in our news section: curesma.org/scholar-rock-a…

Streamers @sim_simmy 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!

Join streamer @sim_simmy 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

Scholar Rock is proud to be a Title Sponsor of the @CureSMA 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

After bringing his story to the University of California Board of Regents, Ryan Manriquez, adult with SMA and UC Berkeley student, has prompted the UC to ensure emergency evacuation chairs are in every multi-storied building in the 10-university system. Full story below

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

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…