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Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado

May 21, 2019
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Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to […]

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Novartis Issues Community Statement on Branaplam Clinical Study

May 16, 2019
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Dear SMA Community, We are writing to inform you that enrollment to the ongoing branaplam clinical study is now closed. Branaplam is currently being assessed […]

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Team Biogen Runs from Chicago to Madison, Raising Over $12,000 for Cure SMA

May 16, 2019
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On Friday, May 17th to Saturday, May 18th, Team Biogen will hit the ground running in the 2019 Reebok Ragnar Great Midwest, a 14-person relay […]

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Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA

May 14, 2019
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Your voice has been heard! Cure SMA has published a paper, titled, Evaluating Benefit-Risk Decision-Making in Spinal Muscular Atrophy: A First-Ever Study to Assess Risk Tolerance […]

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Spring 2019 SMA Newborn Screening Update

May 13, 2019
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We are thrilled to announce that Missouri, Pennsylvania and Vermont have now implemented permanent statewide SMA newborn screening, making 6 states that are now permanently […]

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Team Cure SMA to Participate in Vermont City Marathon

May 9, 2019
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On Sunday, May 26th, Regina Philipps will participate in the Vermont City Marathon and has already raised over $13,000 for Team Cure SMA while training. […]

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Dr. Arthur Burghes Named 2019 Distinguished Scholar

May 8, 2019
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The Ohio State University recently announced the 2019 honorees of the Distinguished Scholar Award. The award, established in 1978, recognizes exceptional scholarly accomplishments by senior […]

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Scholar Rock Issues Community Statement on the Initiation of a New Clinical Trial for SRK-015

May 8, 2019
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The below statement is a community update from Scholar Rock on SRK-015.  Dear SMA Community, Scholar Rock is dedicated to developing novel medicines to improve […]

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Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the AAN Annual Meeting

May 7, 2019
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Genentech, a member of the Roche Group, recently announced new data at the 71st American Academy of Neurology (AAN) Annual Meeting from the dose-finding Part […]

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AveXis Presents Data at AAN Annual Meeting Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients

May 6, 2019
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AveXis, a Novartis company, recently announced interim data from ongoing trials of the investigational product Zolgensma® (onasemnogene abeparvovec-xioi; AVXS-101) that showed positive results across a […]

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