B4GALT1-congenital disorders of glycosylation presents as a non-neurologic glycosylation disorder with hepatointestinal involvement.

Guillard, Maïlys; Morava, Eva; de Ruijter, Jorg; Roscioli, Tony; Penzien, Johann; van den Heuvel, Lambert; Willemsen, Michel A; de Brouwer, Arjan et al. · J Pediatr · 2011

case_report · Level V

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Abstract

The clinical phenotype of congenital disorders of glycosylation is heterogeneous, mostly including a severe neurological involvement and multisystem disease. We identified a novel patient with a galactosyltransferase deficiency with mild hepatopathy and coagulation anomalies, but normal psychomotor development. The tissue-specific expression of the defective B4GALT1 gene correlated with the clinical phenotype.

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