Fabry disease: current treatment and future perspective
Author:
Affiliation:
1. Department of Pediatrics, Center for Genomic Medicine, Bundang CHA Medical Center, CHA University School of Medicine, Seongnam, Korea
Publisher
Korean Society of Medical Genetics
Subject
General Medicine
Link
http://pdf.medrang.co.kr/JGM/2023/020/jgm-20-1-6.pdf
Reference46 articles.
1. Fabry disease revisited: Management and treatment recommendations for adult patients
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3. Newborn screening for lysosomal storage disorders
4. Fabry Disease: prevalence of affected males and heterozygotes with pathogenic GLA mutations identified by screening renal, cardiac and stroke clinics, 1995–2017
5. Natural course of Fabry disease and the effectiveness of enzyme replacement therapy: a systematic review and meta-analysis
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1. Fabry disease management: Current status, therapeutic challenges, and future horizons in drug delivery and artificial intelligence assisted diagnosis;Journal of Drug Delivery Science and Technology;2024-10
2. Restoration of peripheral neuropathy in Fabry mice via intrathecal administration of an adeno-associated virus vector encoding mGLA cDNA;Molecular Genetics and Metabolism;2024-09
3. Cardiac manifestations of Fabry disease in G3Stg/GlaKO and GlaKO mouse models–Translation to Fabry disease patients;PLOS ONE;2024-05-31
4. Inflammation and Exosomes in Fabry Disease Pathogenesis;Cells;2024-04-09
5. Modern Approach to Fabry Disease Diagnosis and Management in Children;Current Pediatrics;2024-02-25
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