A phase 3 randomized, double blind, placebo-controlled study to evaluate the efficacy and safety of sialic acid extended-release tablets in patients with GNE myopathy (GNEM)


GNE Myopathy (GNEM), also known as hereditary inclusion body myopathy (HIBM), is a severe progressive adult-onset myopathy caused by bi-allelic mutations of the GNE gene resulting in a defect in the sialic acid biosynthetic pathway. Aceneuramic acid extended release (Ace-ER) is being developed with the goal of replacing the deficient sialic acid. A phase 2 study demonstrated stabilization in the upper extremity (UE) with Ace-ER 6g/day over a 12-month period. This pivotal Phase 3 study is being conducted to further evaluate the efficacy and safety of Ace-ER 6g/day.


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