Rare Disease Day

Ultragenyx Announces Closure of Clinical Trial for GNE Myopathy

2017-08-23

http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=1037951

"a Phase 3 study evaluating aceneuramic acid extended release (Ace-ER) in patients with GNE Myopathy (GNEM) did not achieve its primary endpoint of demonstrating a statistically significant difference in the upper extremity muscle strength composite score compared to placebo. The study also did not meet its key secondary endpoints.". For details read 

http://www.genengnews.com/gen-news-highlights/ultragenyx-halts-ace-er-development-after-phase-iii-failure/81254845