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GALK1 deficiency galactosemia in vivo galactose oxidation stable‐isotope labeled galactose

Journal

JIMD reports
ISSN: 2192-8304
Titre abrégé: JIMD Rep
Pays: United States
ID NLM: 101568557

Informations de publication

Date de publication:
May 2021
Historique:
received: 11 10 2020
revised: 17 01 2021
accepted: 19 01 2021
entrez: 12 5 2021
pubmed: 13 5 2021
medline: 13 5 2021
Statut: epublish

Résumé

Galactokinase deficiency is an inborn error of carbohydrate metabolism due to a block in the formation of galactose-1-phosphate from galactose. Although the association of galactokinase deficiency with formation of cataracts is well established, the extent of the clinical phenotype is still under investigation. We describe a 6-year-old female who was diagnosed with galactokinase deficiency due to cataract formation when she was 10 months of age and initially started on galactose-restricted diet at that time for 5 months. She developed gait abnormality at 4 years of age. Breath tests via measurement of

Identifiants

pubmed: 33977035
doi: 10.1002/jmd2.12205
pii: JMD212205
pmc: PMC8100398
doi:

Types de publication

Journal Article

Langues

eng

Pagination

104-109

Informations de copyright

© 2021 The Authors. JIMD Reports published by John Wiley & Sons Ltd on behalf of SSIEM.

Déclaration de conflit d'intérêts

The authors have nothing to declare.

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Auteurs

Can Ficicioglu (C)

Department of Pediatrics, Section of Biochemical Genetics The Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine Philadelphia Pennsylvania USA.

Didem Demirbas (D)

Division of Genetics and Genomics, The Manton Center for Orphan Disease Research Boston Children's Hospital, Harvard Medical School Boston Massachusetts USA.

Britt Derks (B)

Department of Pediatrics Maastricht University Medical Centre Maastricht The Netherlands.
Department of Clinical Genetics Maastricht University Medical Centre Maastricht The Netherlands.

G Shashidhar Pai (GS)

Medical University of South Carolina Children's Health, Division of Genetics Charleston South Carolina USA.

David J Timson (DJ)

School of Pharmacy and Biomolecular Sciences University of Brighton Brighton UK.

Maria Estela Rubio-Gozalbo (ME)

Department of Pediatrics Maastricht University Medical Centre Maastricht The Netherlands.
Department of Clinical Genetics Maastricht University Medical Centre Maastricht The Netherlands.

Gerard T Berry (GT)

Division of Genetics and Genomics, The Manton Center for Orphan Disease Research Boston Children's Hospital, Harvard Medical School Boston Massachusetts USA.

Classifications MeSH