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  • amyotrophic lateral s
  • Differential diagnoses include amyotrophic lateral sclerosis, congenital muscular dystrophies, congenital myopathies, primary lateral sclerosis, myasthenia gravis, and carbohydrate metabolism disorders (see these terms). (nih.gov)
  • Clinical
  • Publications] Shiraiwa Y, Saito K et al: 'Clinical features and molecular generic diagnosis of proximal spinal muscular atrophy in childhood'J. (nii.ac.jp)
  • CAMBRIDGE, Mass. & CARLSBAD, Calif.--( BUSINESS WIRE )--New data from the clinical program for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), were presented by Biogen (NASDAQ: BIIB) and Ionis Pharmaceuticals (NASDAQ: IONS) in the late-breaking session at the 2016 World Muscle Society Congress in Granada, Spain. (businesswire.com)
  • New SPINRAZA data continue to reinforce the positive results seen in clinical studies and in my own practice," said Thomas Crawford, M.D., co-director, Muscular Dystrophy Association Clinic at Johns Hopkins Medicine. (biogen.com)
  • contractures
  • This review focuses on orthopedic management of the disease, with discussion of key aspects that include scoliosis, muscular contractures, hip joint disorders, fractures, technical devices, and a comparative approach of conservative and surgical treatment. (dovepress.com)
  • muscles
  • The adult-onset form (sometimes classified as a late-onset SMA type 3) usually manifests after the third decade of life with gradual weakening of muscles - mainly affects proximal muscles of the extremities - frequently requiring the person to use a wheelchair for mobility. (wikipedia.org)
  • nusinersen
  • CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE) September 26, 2016 --Biogen (NASDAQ: BIIB) and Ionis (NASDAQ:IONS) today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). (drugs.com)
  • progressive
  • Soon after, Professor Johann Hoffmann from Heidelberg University presented a paper describing a syndrome of progressive atrophy, weakness, and death during the early childhood period of siblings with genetically normal parents. (medscape.com)