Research
2016 Research Year-in-Review: An Historic Year for Our Community
This has been an historic year for the SMA community. On December 23, the FDA announced its approval of Spinraza, the first-ever approved treatment for […]
Read More ›The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants
Today, Lancet published results from the Phase 2, open-label trial of nusinersen (Spinraza) in infants. This trial initiated in 2013, and patients continue to receive […]
Read More ›Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on Clinical Trial Outcomes in Adults with SMA
Cure SMA-funded researcher, Chad Heatwole, and his team, have published a paper, “What Matters Most: A Perspective From Adult Spinal Muscular Atrophy Patients”. This project […]
Read More ›Over 35 Cure SMA-Funded Researchers Contribute to Comprehensive SMA Encyclopedia
Spinal Muscular Atrophy: Disease Mechanisms and Therapy, the first comprehensive encyclopedia of SMA, has been published by Elsevier. Over thirty five of the book’s authors […]
Read More ›Registration is Now Open for the 2017 SMA Researcher Meeting
Registration is now open for industry and academic researchers to attend our 2017 SMA Researcher Meeting, which will be held June 29-July 1 at Disney […]
Read More ›Spotlight Innovation Launches Development of STL-182 to Treat SMA
Spotlight Innovation has obtained an exclusive, worldwide license to STL-182, a novel compound invented by Drs. Elliot Androphy at Indiana University and Kevin Hodgetts, director […]
Read More ›Cure SMA-funded Researcher Sara Custer Publishes Paper
Sara Custer, PhD, and her colleagues at Indiana University have published a new paper, “Altered mRNA Splicing in SMN-Depleted Motor Neuron-Like Cells” in the journal […]
Read More ›Roche Launches New Phase 2 Clinical Trials
Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in October. Earlier this month, […]
Read More ›Catch Up on Research News from the 2016 Family Friendly Research Poster Session
Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show promise in […]
Read More ›Roche Provides New Update on Clinical Development of RG7916
Roche, PTC Therapeutics, and the SMA Foundation have released a community update on the development of their SMN2 splicing modifier clinical development program: Dear SMA […]
Read More ›