Latest News
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 pivotal global study evaluating the […]
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 regarding timing of treatment initiation, […]
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 week of workshops, networking, and […]
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 atrophy (SMA) being added to […]
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). New late-breaker data from the […]
Read More ›Financial Planning with a Disability
When you first learned that you or your child had SMA, you probably began analyzing your home life, your or your child’s education, and other immediate concerns. In time, you […]
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