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The effects of transitioning from immediate release to extended release cysteamine therapy in Norwegian patients with nephropathic cystinosis: a retrospective study

BACKGROUND: Nephropathic cystinosis is a rare lysosomal storage disorder in which accumulation of cystine and formation of crystals particularly impair kidney function and gradually lead to multi-organ dysfunction. Lifelong therapy with the aminothiol cysteamine can delay the development of kidney f...

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Detalles Bibliográficos
Autores principales: Bjerre, Anna, Aase, Sonja Amdal, Radtke, Maria, Siva, Christian, Gudmundsdottir, Helga, Forsberg, Brita, Woldseth, Berit, Brackman, Damien
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10514171/
https://www.ncbi.nlm.nih.gov/pubmed/37219641
http://dx.doi.org/10.1007/s00467-023-06005-w