Macular Hypoplasia in Congenital Disorder of Glycosylation Type Ia

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Macular Hypoplasia in Congenital Disorder of Glycosylation Type Ia

Congenital disorders of glycosylation are a rare group of metabolic disorders that can result in multiorgan disease. This article describes a novel finding of macular hypoplasia in congenital disorders of glycosylation type Ia.

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Prepubertal growth in congenital disorder of glycosylation type Ia (CDG-Ia).

AIMS To delineate the pattern of growth in prepubertal children with congenital disorder of glycosylation type Ia (CDG-Ia) in order to identify critical period(s) and possible cause(s) of growth failure. METHODS Longitudinal measurements of weight, length/height, and head circumference from birth to 10 years of age in 25 CDG-Ia patients with the R141H/F119L PMM2 genotype were analysed. The da...

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Congenital disorder of glycosylation type Ia (CDG-Ia): phenotypic spectrum of the R141H/F119L genotype.

AIMS To delineate common and variable features and outcome of children with congenital disorder of glycosylation type Ia (CDG-Ia) caused by the frequent R141H/F119L PMM2 genotype. METHODS Clinical data on 25 patients (mean age 7.6 years, range 0-19) were analysed. RESULTS All patients had an early presentation with severe feeding problems and failure to thrive, hypotonia, hepatic dysfunctio...

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Congenital disorder of glycosylation type Ia: a non-progressive encephalopathy associated with multisystemic involvement.

Dr. Jaime Moritz Brum – Laboratório de Genética Bioquímica 00SMHS Quadra 501 Bloco A 70335-901 Brasília DF Brasil. E-mail: [email protected] Congenital disorders of glycosylation (CDG) are a group of severe, autosomal recessive, multisystemic diseases, characterized by abnormal glycosylation of glycoproteins and glycolipids. The first disease of this group was reported in 1980. Since then, at leas...

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Leukocyte phosphomannomutase activity in diagnosis of congenital disorder of glycosylation Ia.

Phosphomannomutase (PMM; EC 5.4.2.8) is a cytosolic enzyme that catalyzes the reversible conversion of mannose 6-phosphate to mannose 1-phosphate, a substrate for the synthesis of GDP-mannose, which is a nucleotide sugar required in glycosylation. Previously known in yeast (1, 2), Van Schaftingen and Jaeken (3 ) described the enzyme in 1995 in humans and its deficiency in patients with congenit...

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ژورنال

عنوان ژورنال: Case Reports in Ophthalmology

سال: 2012

ISSN: 1663-2699

DOI: 10.1159/000338627