A Phase 2 Open-label Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy (HIBM)) Patients With Severe Ambulatory Impairment

Status: Discontinued

Phase of Trial: Phase II

Latest Information Update: 06 Nov 2021

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At a glance

Drugs Aceneuramic acid (Primary)
Indications Nonaka distal myopathy
Focus Adverse reactions
Sponsors Ultragenyx Pharmaceutical

Most Recent Events

01 Jun 2018 Status changed from active, no longer recruiting to discontinued.
28 Jan 2018 The study has been discontinued in Bulgaria.
14 Sep 2017 Status changed from recruiting to active, no longer recruiting.

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