Clinical Trial: Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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Official Title: 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) or Hereditary Inclusion Bo

Brief Summary: UX001-CL301 is a randomized, double-blind, placebo-controlled study to assess the safety and efficacy of 6 g/day sialic acid - extended release (SA-ER) treatment as compared with placebo in subjects with GNE Myopathy.