Research
Biogen Shares SPINRAZA Research Update
New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of SMA Patients Biogen Inc. recently announced additional data from the SPINRAZA […]
Biogen Releases Q2 Community Statement on Latest Milestones in Clinical Development Program
Dear Members of the SMA Community, In light of the COVID-19 pandemic and the burden it has placed on our healthcare system, Biogen is working […]
Update from Scholar Rock on TOPAZ Phase 2 Clinical Trial of SRK-015 in Patients with SMA
Scholar Rock recently reported financial results for the first quarter ending March 31, 2020, highlighting progress and upcoming milestones for its pipeline programs—include the SRK-015 […]
Genentech’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants With Type 1 SMA
Genentech, a member of the Roche Group, today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged […]
Annual Cure SMA Community Update Survey Ready for Your Input
Navigating the recent global health crisis, Coronavirus (COVID-19), has reinforced for us the importance of understanding what the SMA community is thinking, feeling, and experiencing. […]
Grant Announcement: Cure SMA Awards $190,000 Grant to Stephen Meriney, Ph.D., University of Pittsburgh
This article is the last in a series of Cure SMA grant announcements that have been shared throughout the past months. Cure SMA has awarded […]
Biogen Announces First Patient Treated With Higher Dose Of Spinraza In Phase 2/3 DEVOTE Study
Biogen today announced that the first patient has been treated in the global clinical study, DEVOTE. The study is designed to evaluate the safety, tolerability, […]
Zolgensma® Data Shows Rapid, Significant, Clinically Meaningful Benefit in SMA, Including Prolonged Event-free Survival, Motor Milestone Achievement and Durability, Now Up to 5 Years Post-dosing
AveXis, a Novartis company, announced a one-time infusion of Zolgensma® (onasemnogene abeparvovec-xioi) showed rapid, significant, and clinically meaningful therapeutic benefit in patients with spinal muscular […]
Cure SMA Launches Part 2 of “Spotlight on SMA” in Partnership with Neurology Reviews
Cure SMA is pleased to announce the release of “Spotlight on SMA: The Spinal Muscular Atrophy Treatment Landscape,” the final supplement in a two-part series […]
Grant Announcement: Cure SMA Awards $150,000 Grant to Emily Welby, Ph.D., Medical College of Wisconsin
This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA has awarded a $150,000 grant to […]