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Treatment Options in Congenital Disorders of Glycosylation
- Source :
- Frontiers in Genetics, Frontiers in Genetics, Vol 12 (2021)
- Publication Year :
- 2021
- Publisher :
- Frontiers Media S.A., 2021.
-
Abstract
- Despite advances in the identification and diagnosis of congenital disorders of glycosylation (CDG), treatment options remain limited and are often constrained to symptomatic management of disease manifestations. However, recent years have seen significant advances in treatment and novel therapies aimed both at the causative defect and secondary disease manifestations have been transferred from bench to bedside. In this review, we aim to give a detailed overview of the available therapies and rising concepts to treat these ultra-rare diseases.
- Subjects :
- Glycosylation
glycosylation
Disease
Review
QH426-470
Bioinformatics
substrate supplementation
chemistry.chemical_compound
congenital disorder of glycosylation
medicine
Genetics
chaperone
Genetics (clinical)
treatment
drug repurposing
business.industry
Secondary disease
Treatment options
cofactor
medicine.disease
Drug repositioning
chemistry
Chaperone (clinical)
Molecular Medicine
Identification (biology)
business
Congenital disorder of glycosylation
Subjects
Details
- Language :
- English
- ISSN :
- 16648021
- Volume :
- 12
- Database :
- OpenAIRE
- Journal :
- Frontiers in Genetics
- Accession number :
- edsair.doi.dedup.....ed8ceeeac55df1e80d629afeeb2d208f