Research

Cytokinetics and Astellas Announce New Phase 2 Clinical Trial

December 23, 2014
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Cytokinetics and Astellas jointly announced plans to begin a Phase 2 clinical trial in 2015 in patients with spinal muscular atrophy. The trial will test […]

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Cure SMA Announces New $50,000 Clinical Care Research Grant to Nilesh Mehta at Boston Children’s Hospital

December 19, 2014
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We’re excited to announce that we have awarded $50,000 clinical care grant to Dr. Nilesh Mehta, for his project focusing on nutrition in SMA. Some […]

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Results Published on First Large Animal Model of SMA

December 18, 2014
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Dr. Arthur Burghes, a Cure SMA Scientific Advisory Board Member from Ohio State University, has published a paper on the first-ever large animal model of […]

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Cure SMA Funding Leads to 16 New Research Publications in 2014

December 9, 2014
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An important goal of our research funding strategy is to share scientific findings with the broader scientific community. Scientists who receive Cure SMA funding often […]

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Cure SMA Funds 26 Research Projects in 2014

December 2, 2014
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Our strategic research approach includes four areas—basic research, drug discovery, clinical trials, and clinical care—working together toward our vision of a world without SMA. Cure […]

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Isis Expands Phase 3 Clinical Trials

November 25, 2014
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Isis recently announced that they have tested the first patient in CHERISH, a multi-center Phase 3 clinical study evaluating the efficacy and safety of the […]

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SMA Drug Pipeline Continues to Grow

November 21, 2014
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This is an exciting time for the entire SMA community, as we celebrate another year of increasing momentum toward our goal of a treatment and […]

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Cure SMA Researchers Present at Society for Neuroscience Meeting

November 21, 2014
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The Society for Neuroscience (SfN) Meeting is the premiere neurobiology meeting, with over 30,000 scientists in attendance each year. This year’s meeting was held November […]

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PTC Therapeutics Announces New Phase 1a/2b Clinical Trial

November 19, 2014
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PTC Therapeutics, Inc. announced today that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) has started a […]

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Isis Announces Second Phase III Clinical Trial

November 18, 2014
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Isis recently announced the start of CHERISH, a multi-center Phase III clinical study evaluating the efficacy and safety of the investigational compound, ISIS-SMNRx, in non-ambulatory […]

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