Front Page News
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 […]
Community Spotlight: Nick Farrell
In honor of National Volunteer Month in April, Cure SMA is sharing stories and quotes from volunteers who support our programs and further grow our […]
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 […]
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 […]
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 […]
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 […]
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 […]