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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...
Autores principales: | Bjerre, Anna, Aase, Sonja Amdal, Radtke, Maria, Siva, Christian, Gudmundsdottir, Helga, Forsberg, Brita, Woldseth, Berit, Brackman, Damien |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Berlin Heidelberg
2023
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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 |
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