Rationale and Design for a Phase 1 Study of N -Acetylmannosamine for Primary Glomerular Diseases.

We recently demonstrated that ManNAc was safe and well tolerated in a first-in-human phase 1 study in subjects with UDP--acetylglucosamine (GlcNAc) 2-epimerase/ManNAc kinase (GNE) myopathy, a disorder of impaired sialic acid synthesis. Using previous preclinical and clinical data, we propose to test ManNAc therapy for subjects with primary glomerular diseases. Even though the exact mechanisms, affected cell types, and pathologic consequences of glomerular hyposialylation need further study, treatment with this physiological monosaccharide could potentially replace or supplement existing glomerular diseases therapies.