Cure SMA Awards $300,000 Grant to Kevin Hodgetts, PhD, The Brigham and Women’s Hospital

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Cure SMA has awarded a $300,000 preclinical drug discovery grant to Kevin Hodgetts, PhD, at the Brigham and Women’s Hospital, for his project, ” Pre-Clinical Development of LDN-5178 for the Treatment of SMA.” This grant will be conducted by two academic research teams working together to identify new treatments for SMA. The two teams are […]

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Dr. John Kissel, Chair of Neurology at OSU Wexner Medical Center, Retires After 40 Years

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Dr. John Kissel has treated patients with neuromuscular disorders from all over the United States. He specializes in spinal muscular atrophy and has been instrumental in his practice at one of the country’s leading centers in neuromuscular medicine. Dr. Kissel’s interest in SMA began during his time as a fellow at Ohio State University Hospital

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Roche Releases Community Update on Risdiplam

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The following is a community update from Genentech/Roche on the risdiplam (RG7916) program. Dear SMA advocacy community, As requested, we are very pleased to share an update on the RG7916 program. We would also like to extend a special thanks to study participants and their families for their incredible commitment to advancing the progress of

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Cure SMA Awards $300,000 Grant to Umrao Monani, PhD, Columbia University

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Cure SMA has awarded a $300,000 preclinical drug discovery grant to Umrao Monani, PhD, at Columbia University, for his project, ” Restoring function at the NMJ: A novel means to treat SMA.” A drug directed at SMN enhancement, Spinraza, has recently become available but is unlikely to benefit all patients with maximal effectiveness. The objective

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HHS Secretary, Alex Azar, Recommends Nationwide Newborn Screening for SMA

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Health and Human Services Secretary Alex Azar today approved the recommendation that newborn screening for spinal muscular atrophy be implemented nationwide. This recommendation was issued on February 8, 2018, by the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). With Secretary Azar’s signature, SMA becomes part of the Recommended Uniform Screening Panel (RUSP),

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SMA Newborn Screening Moves Forward in New Jersey

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Earlier this month, the New Jersey State Senate Committee on Health, Human Services, and Senior Citizens unanimously approved S. 974. Introduced by Senator Troy Singleton, the bill would add SMA to the New Jersey’s screening panel, and provide follow up information to the family of an affected newborn. Special thanks to SMA community advocates Denise

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Thank You For a Wonderful 2018 Annual SMA Conference!

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Thank you to everyone who attended the 2018 Annual SMA Conference at the Hilton Anatole Hotel in Dallas, Texas! It was an impactful weekend of community, research and fun. While the final numbers are still being counted, we are happy to announce that, including on-site registration, there were around 1,600 attendees in total – our

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Summer 2018 Issue of Compass Now Available

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The summer 2018 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced ten grants totaling $1,325,000 million for basic research. This issue of Compass introduces our grantees and gives a brief overview of their projects, including how the research will be conducted and what impact the

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PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings

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On Saturday, PTC Therapeutics, Inc. announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy (SMA), at the 22nd Annual SMA Researcher Meeting. The data presented by Dr. Baranello demonstrated that at Day 182, over 90% of the babies

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Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference

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Today, Cytokinetics announced data from the Phase 2 clinical study of reldesemtiv in patients with SMA, presented in an oral presentation by John W. Day, M.D., Ph.D., Professor of Neurology and Pediatrics (Genetics), Stanford University, at the 2018 Annual Cure SMA Conference in Dallas. This hypothesis-generating study met its primary objective to determine potential pharmacodynamic

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