A Phase 1 Randomized, Placebo-Controlled, Double-Blind, Escalating Single-Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ManNAc in Subjects With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

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Trial Profile

A Phase 1 Randomized, Placebo-Controlled, Double-Blind, Escalating Single-Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ManNAc in Subjects With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Completed
Phase of Trial: Phase I

Latest Information Update: 25 Oct 2017

At a glance

  • Drugs DEX M74 (Primary)
  • Indications Nonaka distal myopathy
  • Focus Adverse reactions

    • Most Recent Events

    • 26 Aug 2015 New trial record
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