Successful treatment of carbohydrate deficient glycoprotein syndrome type 1b with oral mannose.

Hendriksz, C J; McClean, P; Henderson, M J; Keir, D G; Worthington, V C; Imtiaz, F; Schollen, E; Matthijs, G et al. · Arch Dis Child · 2001

case_report · Level V

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Abstract

An Asian girl presented with failure to thrive, congenital hepatic fibrosis, protein losing enteropathy, and hypoglycaemia. Phosphomannose isomerase activity in skin fibroblasts was reduced. She is homozygous for a mutation, D131N, in the phosphomannose isomerase gene (PM1), consistent with the diagnosis of carbohydrate deficient glycoprotein syndrome type 1b. She responded to oral mannose treatment.

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