Plasma chitotriosidase enzyme activity as a novel therapeutic monitor for cysteamine treatment in nephropathic cystinosis: A retrospective validation study-Reference-Cited by-同舟云学术

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Plasma chitotriosidase enzyme activity as a novel therapeutic monitor for cysteamine treatment in nephropathic cystinosis: A retrospective validation study

Published:2024-05 Issue:1 Volume:142 Page:108454
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Veys Koenraad,Elmonem Mohamed A.,van den Heuvel Lambert,Gahl William A.,Levtchenko Elena

Publisher

Elsevier BV

Reference18 articles.

1. Cystinosis;Gahl;N. Engl. J. Med.,2002

2. Nephropathic cystinosis in adults: natural history and effects of oral cysteamine therapy;Gahl;Ann. Intern. Med.,2007

3. Cysteamine therapy delays the progression of nephropathic cystinosis in late adolescents and adults;Brodin-Sartorius;Kidney Int.,2012

4. An international cohort study spanning five decades assessed outcomes of nephropathic cystinosis;Emma;Kidney Int.,2021

5. Outcome of infantile nephropathic cystinosis depends on early intervention, not genotype : A multicenter sibling cohort study;Veys;J. Inherit. Metab. Dis.,2023

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