Characterization of Early Disease Status in Treatment-Naive Male Paediatric Patients with Fabry Dise...
Characterization of Early Disease Status in Treatment-Naive Male Paediatric Patients with Fabry Disease Enrolled in a Randomized Clinical Trial
About this item
Full title
Author / Creator
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 and Ramaswami, Uma
Publisher
United States: Public Library of Science
Journal title
Language
English
Formats
Publication information
Publisher
United States: Public Library of Science
Subjects
More information
Scope and Contents
Contents
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.
Males aged 5-18 years with complete α-galactosidase A deficiency, without symptoms of major organ damage, were enrol...
Alternative Titles
Full title
Characterization of Early Disease Status in Treatment-Naive Male Paediatric Patients with Fabry Disease Enrolled in a Randomized Clinical Trial
Authors, Artists and Contributors
Author / Creator
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
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_plos_journals_1982584507
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_1982584507
Other Identifiers
ISSN
1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0124987
