Research Grant: “Evaluation of a Novel Drug Combination Treatment of CF10 and Everolimus for Nephropathic Cystinosis in a New Cystinotic Rat Model.”
The lives of cystinosis patients could be improved by developing 1) a better variant of cysteamine that is more tolerable with fewer side effects and 2) alternative therapies that target other pathways affected in cystinosis, such as autophagy, that are likely to play a role in the kidney failure but are not corrected by cysteamine alone. Towards these goals, Drs. Hollywood and Davidson are using rodent models to test a better variant of cysteamine and to find alternative therapies that target other pathways to improve the lives of patients with cystinosis.
Read their latest Progress Report and Study Lay Abstract.