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Fukutin is a glycosyltransferase that transfers ribitol 5-phosphate from cytidine diphosphate-ribitol to the sugar chains of -dystroglycan 8 , which is necessary for linking basal lamina with the cytoskeletal proteins in skeletal muscle and brain

Most small molecule binders of the oxytocinase subfamily of M1 aminopeptidases, e.g., the phosphinic pseudotripeptides 4346 address the active site and establish strong interactions with the catalytic zinc ion

This works by removing the outer layer of skin or destroying melanin-producing cells

While gene therapy is available for a small subset of patients with specific mutations, developing a therapeutic that broadly targets the cellular stresses that lead to cell death could address a major unmet need

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