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Treatment Options in Congenital Disorders of Glycosylation
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 a...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Frontiers Media S.A.
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8461064/ https://www.ncbi.nlm.nih.gov/pubmed/34567084 http://dx.doi.org/10.3389/fgene.2021.735348 |
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author | Park, Julien H. Marquardt, Thorsten |
author_facet | Park, Julien H. Marquardt, Thorsten |
author_sort | Park, Julien H. |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-8461064 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-84610642021-09-25 Treatment Options in Congenital Disorders of Glycosylation Park, Julien H. Marquardt, Thorsten Front Genet Genetics 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. Frontiers Media S.A. 2021-09-10 /pmc/articles/PMC8461064/ /pubmed/34567084 http://dx.doi.org/10.3389/fgene.2021.735348 Text en Copyright © 2021 Park and Marquardt. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Genetics Park, Julien H. Marquardt, Thorsten Treatment Options in Congenital Disorders of Glycosylation |
title | Treatment Options in Congenital Disorders of Glycosylation |
title_full | Treatment Options in Congenital Disorders of Glycosylation |
title_fullStr | Treatment Options in Congenital Disorders of Glycosylation |
title_full_unstemmed | Treatment Options in Congenital Disorders of Glycosylation |
title_short | Treatment Options in Congenital Disorders of Glycosylation |
title_sort | treatment options in congenital disorders of glycosylation |
topic | Genetics |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8461064/ https://www.ncbi.nlm.nih.gov/pubmed/34567084 http://dx.doi.org/10.3389/fgene.2021.735348 |
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