Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) [Phase 3]

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Summary

Phase 3 trial evaluating Ace-ER in GNEM/HIBM was terminated; results were posted in 2019.

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Diseases / indications

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

Drugs mentioned

• Aceneuramic Acid Extended-Release (Ace-ER) Tablets (Aceneuramic Acid Extended-Release)

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles | Phase 3 | Status: TERMINATED | Results posted: 2019-02-19