[1] Sujay Guha. “Pre-clinical evaluation of cysteamine bitartrate as a therapeutic agent for mitochondrial respiratory chain disease.” Human molecular genetics (2019): 1837–1852. [2] Sabrina Klank. “Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients.” Pharmaceutics 15 7 (2023).
용도
Cysteamine bitartrate (CAS# 27761-19-9) treatment of cystinosis and other metabolic and neurodegenerative diseases.
Mechanism of action
Cysteamine Bitartrate is an aminothiol salt used in the treatment of nephropathic cystinosis. Cysteamine bitartrate depletes cystine in lysosomes by reacting with cysteine and forming a mixed cysteine–cysteamine disulfide that can exit the lysosomes via lysine transporters. This prevents the accumulation of cystine crystals in the lysosomes of patients with cystinosis, which can cause considerable damage and eventual destruction of the cells, particularly in the kidneys[2].
