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Article

Characterization of early disease status in treatment-naive male paediatric patients with Fabry disease enrolled in a randomized clinical trial.

PloS one - 1 Jan 2015

Wijburg Frits A, Bénichou Bernard, Bichet Daniel G, Clarke Lorne A, Dostalova Gabriela, Fainboim Alejandro, Fellgiebel Andreas, Forcelini Cassiano, An Haack Kristina, Hopkin Robert J, Mauer Michael, Najafian Behzad, Scott C Ronald, Shankar Suma P, Thurberg Beth L, Tøndel Camilla, Tylki-Szymańska Anna, Ramaswami Uma

Abstract excerpt

TRIAL DESIGN: This analysis characterizes the degree of early organ involvement in a cohort of oligo-symptomatic untreated young patients with Fabry disease enrolled in an ongoing randomized, open-label, parallel-group, phase 3B clinical trial. METHODS: Males aged 5-18 years with complete α-galac...

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Identifiers

PMID: 25955246
DOI: 10.1371/journal.pone.0124987
PMCID: PMC4425695

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-25955246-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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