Research
Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With SMA
Genentech, a member of the Roche Group, recently announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new […]
Read More ›Cure SMA Releases Updated Drug Pipeline
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 20 active programs, including three approved therapies. 10 pharmaceutical partners. 12 […]
Read More ›Survey Alert: SMA Family Caregivers
Family members of people with SMA play an active role in managing the daily lives of their loved one – from physical tasks like feeding, […]
Read More ›Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of […]
Read More ›Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy
Cure SMA’s Jacqueline Glascock, Director of Research Programs, recently talked with Managed Healthcare Executive about information on the three FDA-approved SMA treatments and the need for […]
Read More ›New Data Presented at World Muscle Society 2021 Virtual Congress
This week, new data was presented at the World Muscle Society 2021 Virtual Congress. Check it out below! Scholar Rock presents two posters […]
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 ›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 […]
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