Cystinosis: Therapy adherence and metabolic monitoring in patients treated with immediate-release cysteamine

BACKGROUND: Cystinosis is a metabolic disease caused by intracellular accumulation of cystine within lysosomes. Development of symptoms can be delayed significantly by a life-long therapy with cysteamine, a drug that enters the lysosome and reacts with cystine thereby enabling its export from the or...

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Detalles Bibliográficos
Autores principales: Linden, Simone, Klank, Sabrina, Harms, Erik, Grüneberg, Marianne, Park, Julien H., Marquardt, Thorsten
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7358454/
https://www.ncbi.nlm.nih.gov/pubmed/32685378
http://dx.doi.org/10.1016/j.ymgmr.2020.100620

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