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Fabry Disease and Its Management: A Literature Analysis

A review was conducted to evaluate interventional therapy for Fabry disease. Fabry disease is a multisystemic X-linked storage disorder that affects the entire body and needs to be treated at an early age. The search was conducted using keywords such as “Fabry disease” and “Management” to review the...

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Detalles Bibliográficos
Autores principales: Besekar, Smruti M, Jogdand, Sangita D, Naqvi, Waqar M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cureus 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10154914/
https://www.ncbi.nlm.nih.gov/pubmed/37153259
http://dx.doi.org/10.7759/cureus.37048
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author Besekar, Smruti M
Jogdand, Sangita D
Naqvi, Waqar M
author_facet Besekar, Smruti M
Jogdand, Sangita D
Naqvi, Waqar M
author_sort Besekar, Smruti M
collection PubMed
description A review was conducted to evaluate interventional therapy for Fabry disease. Fabry disease is a multisystemic X-linked storage disorder that affects the entire body and needs to be treated at an early age. The search was conducted using keywords such as “Fabry disease” and “Management” to review the databases. Seven studies were chosen from the 90 studies, and it was discovered that migalastat and enzyme replacement medication were successful in treating the condition, whereas agalsidase beta failed to have a positive effect on the patient. However, this analysis produced ambiguous conclusions. As only a small number of studies were included in the analysis, additional investigations and evaluations based on randomized controlled trials and case studies are required to determine potential drug-related outcomes. There is a need for future therapeutic research to cure genetically affected illnesses and diseases such as Fabry disease.
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spelling pubmed-101549142023-05-04 Fabry Disease and Its Management: A Literature Analysis Besekar, Smruti M Jogdand, Sangita D Naqvi, Waqar M Cureus Other A review was conducted to evaluate interventional therapy for Fabry disease. Fabry disease is a multisystemic X-linked storage disorder that affects the entire body and needs to be treated at an early age. The search was conducted using keywords such as “Fabry disease” and “Management” to review the databases. Seven studies were chosen from the 90 studies, and it was discovered that migalastat and enzyme replacement medication were successful in treating the condition, whereas agalsidase beta failed to have a positive effect on the patient. However, this analysis produced ambiguous conclusions. As only a small number of studies were included in the analysis, additional investigations and evaluations based on randomized controlled trials and case studies are required to determine potential drug-related outcomes. There is a need for future therapeutic research to cure genetically affected illnesses and diseases such as Fabry disease. Cureus 2023-04-02 /pmc/articles/PMC10154914/ /pubmed/37153259 http://dx.doi.org/10.7759/cureus.37048 Text en Copyright © 2023, Besekar et al. https://creativecommons.org/licenses/by/3.0/This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
spellingShingle Other
Besekar, Smruti M
Jogdand, Sangita D
Naqvi, Waqar M
Fabry Disease and Its Management: A Literature Analysis
title Fabry Disease and Its Management: A Literature Analysis
title_full Fabry Disease and Its Management: A Literature Analysis
title_fullStr Fabry Disease and Its Management: A Literature Analysis
title_full_unstemmed Fabry Disease and Its Management: A Literature Analysis
title_short Fabry Disease and Its Management: A Literature Analysis
title_sort fabry disease and its management: a literature analysis
topic Other
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10154914/
https://www.ncbi.nlm.nih.gov/pubmed/37153259
http://dx.doi.org/10.7759/cureus.37048
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