Research
Genentech Shares New Evrysdi Data on Improved Motor Function and Survival in Babies with SMA Type 1
Genentech, a member of the Roche Group, has shared new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi (risdiplam) […]
Biogen Shares New Spinraza Data Exploring Opportunities to Improve Outcomes for SMA Patients
Biogen has shared new data from the Spinraza (nusinersen) clinical development program aimed at optimizing outcomes for people with SMA and advancing understanding of the […]
Participate in Annual Cure SMA Community Update Survey – Open Now!
Last year presented the us with unique challenges, some we are still managing today. However, it has also reinforced the importance of understanding what the […]
Cure SMA Presents Data on Cure SMA Care Center Network and SMA Clinical Data Registry
Cure SMA’s primary focus is supporting care of the highest value to individuals with SMA. As a result of treatments approved by the U.S. Food […]
Scholar Rock Community Statement on TOPAZ 12-Month Topline Data
Scholar Rock provided the below community statement on data for apitegromab (SRK-015) following the distribution of this company press release. Dear Members of the SMA […]
Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on SMA-HI, a Clinical Trial Outcome in Adults with SMA
Dr. Chad Heatwole, a Cure SMA-funded researcher, and his team have published a paper in the Journal, Muscle Nerve, titled “The Spinal Muscular Atrophy Health […]
Nusinersen Research Updates Presented by Biogen
Biogen recently presented data about ongoing studies of SPINRAZA (nusinersen) in patients with Spinal Muscular Atrophy. Updates included the NURTURE, ENDEAR, SHINE, DEVOTE, and RESPOND […]
Research Updates from Scholar Rock
Scholar Rock Presents TOPAZ Interim Analysis Data for Apitegromab in SMA Scholar Rock recently shared 6-month interim analysis data from the TOPAZ Phase 2 clinical […]
Evrysdi Research Updates from Genentech
New 2-year data show Evrysdi continues to demonstrate improvement or maintenance of motor function in people aged 2-25 years with Type 2 or 3 SMA […]
Zolgensma Research Updates from Novartis Gene Therapies
New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children, and durability 5+ years post-treatment Novartis Gene Therapies recently shared new […]