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  • Neurology
  • James P. Van Meerbeke, Research Assistant from the lab of Charlotte J. Sumner, M.D., Associate Professor of Neurology and Neuroscience, Johns Hopkins University School of Medicine, will present the abstract titled " The Therapeutics Effects of RG3039 in Severe Spinal Muscular Atrophy - Mice and Normal Human Volunteers ," during "The Future of Neuroscience Conference: Neurologists and Neuroscientists Defining the Next Generation of CNS Therapies," taking place on April 27. (thestreet.com)
  • 2016
  • CHICAGO, October 20, 2016 - The Muscular Dystrophy Association (MDA) and RYR-1 Foundation today announced a partnership aimed at advancing research and clinical care, raising awareness, and improving education of patients, medical professionals and the public about RYR1-related myopathies. (mda.org)
  • CHICAGO, September 28, 2016 - The Muscular Dystrophy Association (MDA) and Target ALS Foundation today announced a partnership aimed at advancing ALS research and therapy development. (mda.org)
  • CHICAGO, Sept. 19, 2016- The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration's decision to grant accelerated approval for eteplirsen , the first disease-modifying drug to treat the most common childhood form of muscular dystrophy. (mda.org)
  • nusinersen
  • Spinal muscular atrophy type 1 is a devastating disease, and it is encouraging to see that nusinersen may also help people who are at a later stage in the disease process," Servais said. (ahealthyme.com)
  • This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA). (clinicaltrials.gov)
  • infants
  • This week, AveXis, a Novartis company, reported that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) requesting approval of AVXS-101 to treat infants with spinal muscular atrophy (SMA) type 1. (mda.org)
  • Infants with spinal muscular atrophy may require more frequent visits. (chkd.org)
  • gene
  • Today, Novartis, the parent company of AveXis Inc., announced that the U.S. Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) under Priority Review for Zolgensma (formerly known as AVXS-101), a gene therapy for the treatment of spinal muscular atrophy (SMA) type 1. (mda.org)
  • Cord
  • It attacks nerve cells in the spinal cord, leading to muscle weakness, which can affect breathing, swallowing, walking and head control. (ahealthyme.com)
  • Motor neurons are responsible for receiving the nerve impulses transmitted from the brain to the spinal cord and then transmitting those impulses via the peripheral nerves to the muscle. (pdr.net)