We are a team of committed scientists and drug developers whose mission is to develop treatment options for patients and families that have no therapeutic alternatives. We are experts in all aspects of orphan drug research and development.
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Congenital disorders of glycosylation are rare inborn errors of metabolism in which glycosylation (sugar coating) of a variety of tissue proteins and/or lipids is deficient or defective. They can cause serious, sometimes fatal, malfunction of multiple organ systems in affected individuals.
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