Research

Cure SMA Advocates for SMA Community at ICER Hearing

March 8, 2019
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On Thursday, March 7, Cure SMA testified on behalf of the SMA community at a meeting of the Institute for Clinical and Economic Review (ICER). […]

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Biogen Provides 2019 Community Update on Spinraza

March 5, 2019
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Dear members of the SMA community, As we celebrate Rare Disease Day 2019, we are honored to continue supporting the spinal muscular atrophy (SMA) community. […]

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Genentech/Roche and Cure SMA to Co-Host Webinar

March 1, 2019
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On Monday, March 11th, at 12pm CST (10am PST/11am MST/1pm EST) Genentech/Roche and Cure SMA will co-host a webinar for the SMA community. During the […]

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Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans

February 26, 2019
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Scholar Rock today announced positive interim results from the ongoing Phase 1 single- and multiple-ascending dose clinical trial of SRK-015 in healthy adult volunteers. SRK-015, […]

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Cure SMA Awards $150,000 Grant to Veronica Pessino, PhD, Salk Institute for Biological Sciences

February 22, 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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Cure SMA Awards $150,000 Grant to Laxman Gangwani, PhD, Texas Tech University

February 11, 2019
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Cure SMA has awarded a $150,000 research grant to Laxman Gangwani, PhD, at the Texas Tech University Health Sciences Center, for his project entitled, “Function […]

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Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA

January 22, 2019
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Cytokinetics, Incorporated (CYTK) today announced that it has received feedback from the U.S. Food and Drug Administration (FDA) that the Six Minute Walk Test (6MWT) […]

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Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam

January 7, 2019
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On January 7th, PTC Therapeutics, Inc. provided a corporate update, which will be detailed as part of the company’s presentation at the 37th Annual J.P. […]

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Teen and Young Adult Survey Extended Through January 21st

January 7, 2019
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Are you or a family member between the ages of 12 and 25? Do you want to share your opinion on living with SMA and […]

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Cure SMA Announces Expanded Phase 3 of SMA Industry Collaboration

January 2, 2019
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Cure SMA is pleased to announce the launch of an expanded Phase 3 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted […]

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