Research

Roche Releases Community Statement on RG7916 and Olesoxime Programs

March 15, 2018
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Roche has provided the following community statement on RG7916 and Olesoxime investigational programs. Dear SMA Community, We are very pleased to share an update of […]

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Cure SMA Awards $75,000 Grant to Robert Kalb, MD, Northwestern University

March 14, 2018
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Cure SMA has awarded a $75,000 research grant to Robert Kalb, MD, at Northwestern University Feinberg School of Medicine, for his project, “Effects of diminished […]

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Cure SMA Awards $150,000 Grant to Umrao Monani, PhD, Columbia University

March 13, 2018
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Cure SMA has awarded a $150,000 research grant to Umrao Monani, PhD, at Columbia University, for his project, “SMA modulators as a means to revealing […]

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Biogen Announces New Interim Phase 2 Results From NURTURE

March 12, 2018
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Biogen recently announced new interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of SPINRAZA or nusinersen among […]

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Cure SMA Launches Second Annual Community Survey to Address Important Issues in SMA Treatment

March 12, 2018
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Dear Members of the SMA Community, For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, […]

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Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, Medical College of Wisconsin

March 7, 2018
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Cure SMA has awarded a $150,000 research grant to Allison Ebert, PhD, at the Medical College of Wisconsin, for her project, “Role of astrocyte-produced miR-146a […]

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Phase 2 Clinical Trial Results for CK-2127107 Expected in Second Quarter of 2018

February 21, 2018
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Cytokinetics is currently conducting a Phase 2 clinical trial of CK-2127107 – now referred to by the generic name, reldesemtiv – which is designed to […]

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Biogen Releases Statement on CHERISH Data and Plans for New Clinical Research

February 14, 2018
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Biogen has provided the following community statement on the final results from CHERISH, a Phase 3 study of SPINRAZA.  Dear Members of the SMA community, […]

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Winter 2018 Compass Now Available Online

February 9, 2018
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The winter 2018 issue of Compass is now available online. This issue covers Cure SMA’s community survey, and reviews how data is used to improve […]

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Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy

January 30, 2018
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PTC Therapeutics, Inc. recently announced the presentation of early interim data from Part 1, the dose-finding portion of the FIREFISH study. FIREFISH is a two-part […]

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