Front Page News
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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›Biogen SMA Q1 2021 Community Statement
Dear Members of the SMA Community, Each year, the entire rare disease community comes together on the last day of February to […]
Read More ›Community Spotlight: I am…Viola Dwyer
February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative—we have been posting stories about SMA community members, showcasing their […]
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