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Article

Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors.

PloS one - 1 Jan 2017

Arends Maarten, Biegstraaten Marieke, Hughes Derralynn A, Mehta Atul, Elliott Perry M, Oder Daniel, Watkinson Oliver T, Vaz Frédéric M, van Kuilenburg André B P, Wanner Christoph, Hollak Carla E M

Abstract excerpt

Despite enzyme replacement therapy, disease progression is observed in patients with Fabry disease. Identification of factors that predict disease progression is needed to refine guidelines on initiation and cessation of enzyme replacement therapy. To study the association of potential biochemical and clinical prognostic factors with the disease course (clinical events, progression of cardiac and renal disease)...

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Identifiers

PMID: 28763515
DOI: 10.1371/journal.pone.0182379
PMCID: PMC5538714

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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