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TREATMENT OF CONGENITAL DISORDERS OF GLYCOSYLATION (CDG) USING MANNOSE
TREATMENT OF CONGENITAL DISORDERS OF GLYCOSYLATION (CDG) USING MANNOSE
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机译:甘露糖原糖治疗先天性糖基化疾病(CDG)
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摘要
The invention relates to mannose 1-phosphate derivatives and to pharmaceutical or dietary agents, which contain the same and which are used for treating disorders of glycosylation and, in particular, for treating CDG-1a. These derivatives comprise the following general formula (I), wherein: R1 and R2, which can be identical or different, represent H or formula (II); or R1 and R2, together, represent formula (III) with the provision that only one of the radicals R1 and R2 can represent H, and; R3 and R4, which can be identical or different, represent (a), (b), (c) and (d), and radical R3 can also represent H. Aryl represents an aromatic hydrocarbon radical, which can be substituted by a radical alkyl, and alkyl represents a linear or branched, saturated hydrocarbon radical having 1 to 20 carbon atoms.
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