Cure SMA Funding Leads to 16 New Research Publications in 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 publish their findings in peer-reviewed journals. This means that other scientists can learn from their results, which will pay dividends across the wider landscape of SMA research—allowing us to multiply […]

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Hope on the Hill Advances Key SMA Legislative Priorities

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This year’s 4th Annual Hope on the Hill Congressional Dinner in Washington D.C. was a great success. Building on of this year’s Annual SMA Conference—also held in Washington D.C.—the Hope on the Hill Dinner brought together families, government, and industry leaders to work on shared goals like increasing awareness, advancing treatments for SMA, and improving

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Cure SMA Funds 26 Research Projects in 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 SMA has invested $57 million in research since 1984, with $35 million in the past decade alone. In 2014, we provided funding to 26 different research projects across these four

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Isis Expands Phase 3 Clinical Trials

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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 investigational compound, ISIS-SMNRx, in non-ambulatory children with spinal muscular atrophy (SMA). The study is a double-blind, randomized, sham-procedure controlled study in approximately 117 children with SMA at study centers in

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Cure SMA Researchers Present at Society for Neuroscience Meeting

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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 15-19 in Washington D.C. Two dozen presentations on spinal muscular atrophy were given at the meeting, several of which were by scientists funded by Cure SMA. Cure SMA-funded researchers Drs.

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

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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 Phase 1b/2a study in adult and pediatric patients. The placebo-controlled, randomized, multiple-dose study will enroll approximately 48 patients with SMA. Because of a mutation in the survival motor neuron gene

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Isis Announces Second Phase III Clinical Trial

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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 children with Spinal Muscular Atrophy (SMA). The study is a double-blind, randomized, sham-procedure controlled study in approximately 117 children with SMA at study centers in the US, Canada, Europe and

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Southern California Gala of Hope Brings Community and Industry Partners Together to Raise $80,000

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On November 15, 140 guests gathered for the Inaugural Southern California Cure SMA Gala of Hope. This fantastic evening raised over $80,000 in support of Cure SMA. Our thanks to those that attended, donated and supported this event. Special thanks to the event committee—Liz Bahrenburg, Nicole Lomonaco-Sunde, Nikki McIntosh, and Autumn Montoya—for all their hard

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