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Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients

Cystinosis is a severe inherited metabolic storage disease caused by the lysosomal accumulation of cystine. Lifelong therapy with the drug cysteamine bitartrate is necessary. Cysteamine cleaves intralysosomal cystine, and thereafter, it can exit from the organelle. The need for frequent dosing every...

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Detalles Bibliográficos
Autores principales: Klank, Sabrina, van Stein, Christina, Grüneberg, Marianne, Ottolenghi, Chris, Rauwolf, Kerstin K., Grebe, Jürgen, Reunert, Janine, Harms, Erik, Marquardt, Thorsten
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10385520/
https://www.ncbi.nlm.nih.gov/pubmed/37514038
http://dx.doi.org/10.3390/pharmaceutics15071851