GNE and myopathy: In mice, GNE protein expresses at an early embryonic stage and Gne−/− mice is lethal.4, 24 This is consistent with the lack of biallelic null mutations and only “mildly deleterious” mutations reported in GNE myopathy patients.4 Given that Gne‐deficient hyposialylation is the core pathogenic factor, experimental prophylactic treatments with N‐acetylmannosamine or sialic acid metabolites were performed, and effectively avoiding muscle weakness and atrophy in Gne‐deficient mice models were found.25, 26, 27, 28 This shed a new light on targeted therapy for disease in humans in the near future.