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A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis
BACKGROUND: Nephropathic cystinosis is a rare and severe metabolic disease leading to an accumulation of cystine in lysosomes which especially harms kidney function. A lifelong therapy with the aminothiol cysteamine can delay the development of end-stage renal disease and the necessity of kidney tra...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8438894/ https://www.ncbi.nlm.nih.gov/pubmed/34521447 http://dx.doi.org/10.1186/s13023-021-01991-2 |