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FDA Engagement Update: The Voices of SMA

September 23, 2015
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Thanks to our vibrant community, August was one of our largest and best awareness months ever, with unprecedented levels of public awareness and outreach for SMA. Toward the end of […]

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2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 2

September 22, 2015
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We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers the second […]

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Cure SMA Awards $150,000 to Dr. Lee Rubin at Harvard to Advance a New SMA Drug Discovery Program

September 17, 2015
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Originally published on January 18, 2013. Cure SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program, including drug […]

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Pfizer Licenses Cure SMA Quinazoline Drug Program from Repligen

September 17, 2015
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Originally published on January 3, 2013. Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s spinal muscular atrophy (SMA) […]

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Cure SMA Announces Grant to Cytokinetics for Preclinical Development of Tirasemtiv for Spinal Muscular Atrophy

September 17, 2015
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Originally published on April 4, 2013. Award to support testing of Tirasemtiv in mouse models of Spinal Muscular Atrophy. Cytokinetics, Incorporated (Nasdaq: CYTK) and Cure SMA announced the award of […]

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ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy

September 17, 2015
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Originally published on April 23, 2013. Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced in April the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA). […]

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