Front Page News
Novartis Announces Lift of Partial Clinical Trial Hold, Plans to Initiate a New Study of Intrathecal Zolgensma in Older Patients with SMA
Novartis announced that the U.S. Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may […]
Read More ›Data for Genentech’s Evrysdi Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies with Type 1 SMA
Data from Genentech, a member of the Roche Group, recently announced that the New England Journal of Medicine (NEJM) has published data from FIREFISH Part 2, a […]
Read More ›Cure SMA Convenes Working Group to Discuss Biomarkers for SMA
The advent of three approved SMN-upregulating therapeutics for spinal muscular atrophy has dramatically changed the natural history of the disease. There are, however, many uncertainties […]
Read More ›Thank You for Another Fantastic Virtual SMA Conference!
Cure SMA would like to thank everyone who attended the recent 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting. It was an impactful […]
Read More ›In Just Three Years, Cure SMA Marks Nationwide Milestone for 85% of U.S. Babies Now Screened at Birth for SMA
The organization’s grassroots efforts give thousands of families new hope by securing widespread, state-by-state screening for SMA at birth Within three years of spinal muscular […]
Read More ›Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children
Novartis recently announced data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA). […]
Read More ›Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, announced an oral […]
Read More ›New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA
Genentech, a member of the Roche Group, today announced new interim data from two studies of Evrysdi® (risdiplam)—JEWELFISH and RAINBOWFISH. Genentech leads the clinical development of […]
Read More ›Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference
Biogen Inc., today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of […]
Read More ›Ready to Go! Platform Open for 2021 Virtual SMA Conference Events
Cure SMA is excited to share that the platform for the 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting is […]
Read More ›