New Gene Therapy Results Announced at World Muscle Congress

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Nationwide Children’s Hospital/AveXis recently announced new results from their ongoing Phase 1 trial of systemic gene therapy for SMA. Dr. Jerry R. Mendell, Director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital (NCH), made the presentation at last week’s World Muscle Congress in the United Kingdom. Dr. Mendell, lead […]

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Registration Open for the 2016 SMA Researcher Meeting

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Registration is now open for industry and academic researchers to attend our 2016 SMA Researcher Meeting, which will be held June 16-18 in Anaheim, CA, at the Disneyland Hotel. The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. The 2015 SMA Researcher Meeting set a record, with over

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House of Representatives Passes Ensuring Access to Clinical Trials Act

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The House of Representatives has passed the Ensuring Access to Clinical Trials Act of 2015 (EACT). This bill already passed the Senate in July, and will now go to President Obama to be signed into law. This bill allows individuals to receive up to $2,000 as compensation for participating in a clinical trial, without those

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FDA Engagement Update: The Voices of SMA

Leave a Comment / Advocacy, Community Awareness, Front Page News /

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 August, we challenged our community to seize this momentum, and convert awareness into action and advocacy. One of these opportunities to convert awareness into action

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

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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 half of a session on SMN Partners and Therapeutic Targets. The session was moderated by Rashmi Kothary, PhD. SMN Partners and Candidate Therapeutic Targets, Part

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

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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 discovery programs to make practical new therapies.   Dr. Rubin’s project at Harvard University is the first of three drug discovery awards that will be given

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Pfizer Licenses Cure SMA Quinazoline Drug Program from Repligen

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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) program, originally in-licensed from Cure SMA. Cure SMA funded and directed the preclinical development of RG3039 with an investment of more than $13 million. This

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

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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 a grant from Cure SMA to Cytokinetics to support preclinical research on muscle function in a mouse model of spinal muscular atrophy (SMA) to be

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ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy

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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). The study, which will begin enrolling patients soon, is a Phase 2 study in eight patients with infantile-onset SMA. The study, which initiates the Phase

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