AI Brings Hope for Patients with Lyosomal Storage Disease; Artificial Intelligence Is Becoming Increasingly Important in Drug Discovery. Advances in Use of Big Data, Learning Algorithms, and Powerful Computers Have Now Enabled Researchers at University of Zurich to Better Understand a Serious Metabolic Disease (Cystinosis); Promising Drug Identified

Cystinosis is a rare lyosomal storage disorder affecting approximtely 1 in 100,000 to 200,000 newborns worldwide. Nephropathic (non-inflammatory) cystinosis, the most common and severe form of the disease, manifests with kidney disease symptoms during the first months of life, often leading to kidney failure before the age of 10. “Children with cystinosis suffer from a devastating, multisystemic disease, and there are currently no available curative treatments,” says Olivier Devuyst, MD, PhD, Head of the Mechanisms of Inherited Kidney Disorders (MIKADO) group and Co-Director of the ITINERARE University Research Priority Program at the University of Zurich (UZH).