Research
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 ›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 ›Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM
Genentech, a member of the Roche Group, announced in a press release that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal […]
Read More ›Cure SMA Offer Perspectives on Combination Therapy for SMA
New developments in treatments for spinal muscular atrophy (SMA) over the last few years have changed what is possible for people with SMA, allowing them […]
Read More ›Cure SMA Funds Over $9 Million in New and Ongoing Research in FY20
During our past fiscal year—from July 1, 2019 to June 30, 2020—Cure SMA funded more than $9 million in new and ongoing research and care […]
Read More ›Cure SMA, Novartis Publish Economic Burden of SMA Analysis in Journal of Market Access & Health Policy
Recently, Cure SMA and colleagues at Novartis Gene Therapies published a manuscript titled, “Economic burden of spinal muscular atrophy: an analysis of claims data” in […]
Read More ›