- Enrollment in pivotal Phase 3 study in GNE Myopathy ongoing. The randomized, double-blind, placebo-controlled 48-week study in approximately 80 patients was initiated in May 2015. The study is evaluating the efficacy of Ace-ER with the primary endpoint of a composite of upper extremity muscle strength. Data from the study are expected in the first half of 2017.
Catalyst
Slingshot members are tracking this event:
Phase 3 data of Aceneuramic acid extended release (Ace-ER) for GNE Myopathy due 2H 2017
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Additional Information
Slingshot Insights Explained
Catalyst Date
Occurred on:
Aug 22, 2017
Occurred Source:
http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=1037951
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Related Keywords
Phase 3 Data, Aceneuremic Acid Extended Release, Ace-er, Gne Myopathy
