Clinical Trial Update: A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment [Phase 2]

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Summary

The Phase 2 trial of Aceneuramic Acid Extended Release (Ace-ER; UX001) tablets for patients with GNE myopathy was terminated, with results posted in February 20…

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Diseases / indications

• Gne Myopathy

Drugs mentioned

• Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles | Phase 2 | Status: TERMINATED | Results posted: 2019-02-19