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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Cure SMA Scientific Advisory Board Meets in Washington D.C.

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Today and tomorrow, Cure SMA’s Scientific Advisory Board (SAB) will be meeting in Washington D.C. Our Scientific Advisory Board is a recognized group of experts in SMA and motor neuron biology. They provide strategic and practical guidance in shaping our research programs. This meeting will focus on two main priorities: awarding up to $700,000 in

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Families, Government, and Industry Leaders Unite to Help Cure SMA

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This year’s 4th Annual Hope on the Hill Congressional Dinner in Washington D.C. is less than three weeks away. At Cure SMA, we know that no single group can develop a treatment for SMA alone—it requires collaboration between academics, industry, government, and families. That is why we’re committed to tangible, meaningful efforts to strengthen and

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Medical Advisory Council Gathers for Fall 2014 Meetings

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Members of the Cure SMA Medical Advisory Council (MAC) are holding two days of meetings, November 7-8, at the Cure SMA national offices. The MAC is one of the most highly respected bodies of SMA medical and clinical experts in the US, setting the agenda for proactive, creative, and collaborative leadership on issues that improve

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BAYADA Pediatrics Renews National Sponsorship with Cure SMA

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Cure SMA and BAYADA Pediatrics is proud to jointly announce that BAYADA has renewed its partnership with Cure SMA, as a national sponsor of the Walk-N-Roll program. Throughout the past year, in the true spirit of providing community service where they live and work, BAYADA Pediatrics offices came together to raise funds and awareness for

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Promising Results Published on Cure SMA-Funded Gene Therapy Project

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A manuscript from the laboratory of Dr. Brian Kaspar of Nationwide Children’s Hospital was recently published in the journal Molecular Therapy. The paper, “Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA—a dose response study in mice and nonhuman primates” is the first publication resulting from a groundbreaking collaboration between Cure SMA, the

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Read the Fall 2014 Issue of Compass

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The latest issue of Compass, our research publication, is now available online. This Fall 2014 issue includes news from our 2014 SMA researcher meeting, and news from the 2014 CME conference. Also in this issue, Cure SMA Scientific Advisory Board member Arthur Burghes, PhD, provides an update from our 2014 conference special session, “Moving Beyond

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