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Article

Developments in the treatment of Fabry disease.

Journal of inherited metabolic disease - 1 Sept 2020

van der Veen Sanne J, Hollak Carla E M, van Kuilenburg André B P, Langeveld Mirjam

Abstract excerpt

Enzyme replacement therapy (ERT) with recombinant α-galactosidase A (r-αGAL A) for the treatment of Fabry disease has been available for over 15 years. Long-term treatment may slow down disease progression, but cardiac, renal, and cerebral complications still develop in most patients. In addition, lifelong intravenous treatment is burdensome. Therefore, several new treatment approaches have been explored over the...

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Source

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Identifiers

PMID: 32083331
DOI: 10.1002/jimd.12228
PMCID: PMC7540041

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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