SMA Newborn Screening Guidelines Published in Journal of Neuromuscular Diseases

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The recent decision of the federal Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) to recommend SMA for addition to the RUSP, as well as the implementation of SMA newborn screening (NBS) by several states, has ushered in the ability to identify infants with SMA before the onset of disease symptoms. To aid […]

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Cure SMA Welcomes Back AveXis as a 2018 National Premier Partner

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Cure SMA is excited to announce that AveXis is returning as a National Premier Partner for 2018. As part of their partnership, AveXis is generously supporting our Chapter Leader Summit, a full day of training and information-sharing for 70 chapter leaders from around the US, scheduled for April 2018. Many of the topics and training

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Cure SMA Awards $150,000 Grant to Brunhilde Wirth, PhD, Institute of Cologne, Germany

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Cure SMA has awarded a $150,000 research grant to Brunhilde Wirth, PhD, at Institute of Cologne, Germany, for her project, “Study of combinatorial therapy based on SPINRAZA together with a novel protective genetic modifier.” Based on so-called SMA discordant families, in whom SMN1-deleted individuals remain asymptomatic, Dr. Wirth and her team have recently identified two

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Cure SMA Awards $50,000 Grant to Rashmi Kothary, PhD, University of Ottawa

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Cure SMA has awarded a $50,000 research grant to Rashmi Kothary, PhD, at the University of Ottawa, for his project, ” The shifting landscape of SMA research: towards a better understanding for a role for SMN in aging.” In this proposal, Dr. Kothary and his team seek to explore the role of SMN protein in

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Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with SMA

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Scholar Rock today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead antibody product candidate, SRK-015, for the treatment of spinal muscular atrophy (SMA). SRK-015 is an inhibitor of the activation of myostatin, which Scholar Rock believes has the potential to be the first muscle-directed therapy

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Cure SMA Awards $150,000 Grant to Robin Parks, PhD, University of Ottawa

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Cure SMA has awarded a $150,000 research grant to Robin Parks, PhD, at the University of Ottawa, for his project, “Serum-derived exosomes as a biomarker for Spinal Muscular Atrophy.” Dr. Parks and his team are exploring the use of exosomes, small particles released from all cells, as potential biomarkers for SMA. They have shown that

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Cure SMA Welcomes Back Biogen as a National Premier Partner for 2018

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Cure SMA is thrilled to welcome Biogen back as a National Premier Partner for 2018. As a part of their partnership, Biogen will be sponsoring all walk-n-rolls and galas across the US for the entirety of 2018, as well as the Hope on the Hill Congressional Dinner. Biogen is also a key participant in many

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House and Senate Vote to Increase Federal Funding for Newborn Screening Programs

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Today, Congress passed a new federal spending bill, increasing funding for several critical newborn screening programs, that the President also signed. The bill increases funding for the Health Resources and Services Administration (HRSA) Heritable Disorders Program by more than $2 million, and increases funding for the CDC’s newborn screening quality assurance program by almost $4 million. The

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Cure SMA Awards $150,000 Grant to Charlotte Sumner, MD, Johns Hopkins University

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Cure SMA has awarded a $150,000 research grant to Charlotte Sumner, MD, at Johns Hopkins University, for her project, “Testing the potential of SMN-AS1 as a therapeutic target in SMA.” Dr. Sumner and her team have recently identified a long noncoding RNA (lncRNA) that represses the ability of SMN2 to make its template for protein

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