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01-02-2008 | Brief Report

Intravenous delivery of cysteamine for the treatment of cystinosis: association with hepatotoxicity

Authors: Michael R. Bendel-Stenzel, Julia Steinke, Ranjan Dohil, Youngki Kim

Published in: Pediatric Nephrology | Issue 2/2008

Abstract

Nephropathic cystinosis is a lysosomal storage disorder, which, if untreated, results in renal failure by age 10 years. Oral cysteamine has been shown to preserve renal function in these patients. In this study, a 2-year-old girl with nephropathic cystinosis and severe gastrointestinal dysmotility was treated with intravenous (i.v.) administration of cysteamine hydrochloride (HCl). This is only the second report of long-term i.v. cysteamine therapy for nephropathic cystinosis. Unlike the treatment in the previous case, however, treatment in our patient was limited by liver toxicity.

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Title: Intravenous delivery of cysteamine for the treatment of cystinosis: association with hepatotoxicity
Authors: Michael R. Bendel-Stenzel
Julia Steinke
Ranjan Dohil
Youngki Kim
Publication date: 01-02-2008
Publisher: Springer-Verlag
Published in: Pediatric Nephrology / Issue 2/2008
Print ISSN: 0931-041X
Electronic ISSN: 1432-198X
DOI: https://doi.org/10.1007/s00467-007-0529-8

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Intravenous delivery of cysteamine for the treatment of cystinosis: association with hepatotoxicity

Abstract

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

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