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Autor:
Marquardt, Sabrina Klank, Christina van Stein, Marianne Grüneberg, Chris Ottolenghi, Kerstin K. Rauwolf, Jürgen Grebe, Janine Reunert, Erik Harms, Thorsten
Publikováno v:
Pharmaceutics; Volume 15; Issue 7; Pages: 1851
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=multidiscipl::336cefc1ad8ac133b5485c200ec3d04c
