Research

New Data Presented at World Muscle Society 2021 Virtual Congress

September 24, 2021
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This week, new data was presented at the World Muscle Society 2021 Virtual Congress. Check it out below!     Scholar Rock presents two posters […]

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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

July 29, 2021
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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 […]

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Cure SMA Convenes Working Group to Discuss Biomarkers for SMA

July 1, 2021
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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 […]

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Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children

June 21, 2021
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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). […]

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Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA

June 11, 2021
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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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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

June 11, 2021
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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 […]

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Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference

June 10, 2021
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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 […]

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Cure SMA Publishes Manuscript on SMA Awareness, Screening, and Referral Patterns Amongst Pediatricians

May 28, 2021
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In a May 2021 issue of BMC Pediatrics, Cure SMA published a manuscript, titled “Awareness screening and referral patterns among pediatricians in the United States […]

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Cure SMA-Funded Researchers at Ohio State University Publish Paper on Effect of Nusinersen in Adults with SMA

May 10, 2021
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Dr. Bakri Elsheikh and colleagues at The Ohio State University have published a paper in the Journal, Frontiers in Neurology, titled “Safety, Tolerability, and Effect of Nusinersen […]

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Cure SMA-Funded Researchers at Columbia University Publish Paper on Fatigue in SMA

April 26, 2021
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Dr. Jacqueline Montes and colleagues at Columbia University—part of the Pediatric Neuromuscular Clinical Research Network (PNCRN) funded by Cure SMA—have published a paper in the […]

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