Clinical Trial Update: Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) [Phase 3]

View source ↗

Summary

A Phase 3 randomized, double-blind, placebo-controlled study evaluating sialic acid in GNEM/HIBM (GNE myopathy) has completed with results posted in 2018.

Upgrade for the full analysis, importance score, and drug timeline.

Diseases / indications

• Distal Myopathy Nonaka Type
• Distal Myopathy with Rimmed Vacuoles
• Gne Myopathy
• Hereditary Inclusion Body Myopathy

Drugs mentioned

• Sialic Acid (N-acetylneuraminic acid)

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles | Phase 3 | Status: COMPLETED | Results posted: 2018-07-09