Latest News
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 trial evaluating the potential of […]
Community Statement from Genentech
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 […]
Community Statement from Novartis Gene Therapies
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 […]
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 Genentech, a member of the […]
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 data that reinforce the transformational […]
Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases
Current knowledge regarding clinical meaningfulness and quality of life amongst teens and young adults with spinal muscular atrophy (SMA) is limited. Much of the available qualitative data on this population […]