A phase 3 randomized, double blind, placebo-controlled study to evaluate the efficacy and safety of sialic acid extended-release tablets in patients with GNE myopathy (GNEM) Conference Paper uri icon

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authors

  • Lochmüller, H
  • Behin, A
  • Caraco, Y
  • Lau, H
  • Mirabella, M
  • Tournev, I
  • Tarnopolsky, Mark
  • Pogoryelova, O
  • Shah, J
  • Koutsoubos, T
  • Skrinar, A
  • Mansbach, H
  • Kakkis, E
  • Mozaffar, T

publication date

  • October 2017