Cure SMA is pleased to announce its participation in the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference (March 15-18, 2021) and the American Academy of Neurology (AAN) 2021 Virtual Annual ...
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) in infants aged 1-7 months at enrollment with ...
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 disease. Exploring Opportunities to Optimize Treatment in SMA ...
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 SMA community is thinking, feeling, and experiencing in ...
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 and Drug Administration (FDA), as well as successful ...
Along with funding SMA research and care, Cure SMA is proud to provide thousands of individuals with SMA and their families with vital support they need to navigate life with SMA. In an ...
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 Community, We are pleased to share 12-month topline ...
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 Index (SMA-HI): A Novel Outcome for Measuring How ...
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 trial evaluating the potential of apitegromab, a highly ...
Dear SMA Community, Based on our commitment to the SMA community to provide transparent, timely updates related to our medicines, we are writing to share information regarding recent reports of leaking bottles of ...
Dear Cure SMA Community, Novartis Gene Therapies is committed to patient safety and the ongoing monitoring of adverse events as it relates to the use of ZOLGENSMA® (onasemnogene abeparvovec-xioi). As with all commercially ...
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 Genentech, a member of the Roche Group, recently ...
