Cysteamine bitartrate delayed‐release capsules control leukocyte cystine levels and promote statural growth and kidney health in an open‐label study of treatment‐naïve patients <6 years of age with nephropathic cystinosis

Cysteamine bitartrate delayed‐release capsules control leukocyte cystine levels and promote statural growth and kidney health in an open‐label study of treatment‐naïve patients <6 years of age with nephropathic cystinosis

Abstract Nephropathic cystinosis is a rare autosomal recessive lysosomal storage disease that is characterized by accumulation of cysteine and formation of crystals within cells of different organs and tissues causing systemic manifestations in childhood that include poor linear growth, ocular invol...

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Bibliographic Details
Main Authors: Maria Helena Vaisbich, Juliana Caires Ferreira, Heather Price, Kyleen D. Young, Saba Sile, Gregg Checani, Craig B. Langman
Format: Article
Language:English
Published: Wiley 2022-01-01
Series:JIMD Reports
Subjects:
anthropometric parameters
children
cysteine
cystinosis
delayed‐release cysteamine
immediate‐release cysteamine
Online Access:https://doi.org/10.1002/jmd2.12260

Internet

https://doi.org/10.1002/jmd2.12260

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