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  • Darryl C. De Vivo, Enrico Bertini, Kathryn J Swoboda, Wuh-Liang Hwu, N. Study Group
  • Biology, Medicine
  • Neuromuscular Disorders
  • 2019 (First Publication: 12 September 2019)
  • Spinal muscular atrophy (SMA) is a neurodegenerative disease associated with severe muscle atrophy and weakness in the limbs and trunk. We report interim efficacy and safety outcomes as of March 29,Continue Reading