Research

Genentech’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants With Type 1 SMA

April 28, 2020
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      Genentech, a member of the Roche Group, today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged […]

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Annual Cure SMA Community Update Survey Ready for Your Input

April 23, 2020
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Navigating the recent global health crisis, Coronavirus (COVID-19), has reinforced for us the importance of understanding what the SMA community is thinking, feeling, and experiencing. […]

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Grant Announcement: Cure SMA Awards $190,000 Grant to Stephen Meriney, Ph.D., University of Pittsburgh

April 17, 2020
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This article is the last in a series of Cure SMA grant announcements that have been shared throughout the past months. Cure SMA has awarded […]

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Biogen Announces First Patient Treated With Higher Dose Of Spinraza In Phase 2/3 DEVOTE Study

April 2, 2020
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Biogen today announced that the first patient has been treated in the global clinical study, DEVOTE. The study is designed to evaluate the safety, tolerability, […]

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Zolgensma® Data Shows Rapid, Significant, Clinically Meaningful Benefit in SMA, Including Prolonged Event-free Survival, Motor Milestone Achievement and Durability, Now Up to 5 Years Post-dosing

March 24, 2020
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AveXis, a Novartis company, announced a one-time infusion of Zolgensma® (onasemnogene abeparvovec-xioi) showed rapid, significant, and clinically meaningful therapeutic benefit in patients with spinal muscular […]

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Cure SMA Launches Part 2 of “Spotlight on SMA” in Partnership with Neurology Reviews

March 19, 2020
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Cure SMA is pleased to announce the release of “Spotlight on SMA: The Spinal Muscular Atrophy Treatment Landscape,” the final supplement in a two-part series […]

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Grant Announcement: Cure SMA Awards $150,000 Grant to Emily Welby, Ph.D., Medical College of Wisconsin

March 13, 2020
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This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA has awarded a $150,000 grant to […]

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Biogen Issues Community Letter with Perspective on Coronavirus (COVID-19)

March 11, 2020
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Dear SMA Community, We want to provide an update on Biogen’s efforts to help ensure the health and safety of the SMA community amid the […]

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Grant Announcement: Cure SMA Awards $190,000 Grant to Umrao Monani, Ph.D., Columbia University

March 11, 2020
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This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA has awarded a $190,000 research grant […]

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Cure SMA Awards $100,000 Grant to Rashmi Kothary, Ph.D., Ottawa Hospital Research Institute

March 6, 2020
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This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA and Cure SMA Canada have awarded […]

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