A Phase 1/2, First-in-human, Double-blind, Placebo-controlled Study to Assess Dose, Safety, and Efficacy of UX016 (Sialic Acid-C16 Prodrug) in Adults With GNE Myopathy

Inclusion Criteria: * A confirmed diagnosis of GNEM (also known as hereditary inclusion body myopathy \[HIBM\], distal myopathy with rimmed vacuoles \[DMRV\], inclusion body myopathy 2 \[IBM2\], and Nonaka myopathy in Japan) by Clinical Laboratory Improvement Amendments (CLIA)-certified genetic testing with identification of a disease-associated variant in the gene encoding the GNE/MNK enzyme. Genotyping will not be conducted in this protocol. * Ability to walk a minimum of 20 m independently during Screening. The use of assistive devices and orthotics is allowed. * Has ≤ 80% of normal biceps…