Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients

• Glavni autori: Sabrina Klank (Autor), Christina van Stein (Autor), Marianne Grüneberg (Autor), Chris Ottolenghi (Autor), Kerstin K. Rauwolf (Autor), Jürgen Grebe (Autor), Janine Reunert (Autor), Erik Harms (Autor), Thorsten Marquardt (Autor)
• Format: Knjiga
• Izdano: MDPI AG, 2023-06-01T00:00:00Z.
• Teme: article
• Online pristup: Connect to this object online.

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...