Scientists launch Largest-Ever study to unravel Cystinosis's Long-Term effects

This study follows 250 people with cystinosis, a rare genetic disorder that causes a buildup of the amino acid cystine, leading to damage in the kidneys, eyes, and other organs. Researchers will track how the disease progresses over time, including kidney function, eye problems, and quality of life, while also collecting blood samples for future research. The goal is to better understand the long-term course of the disease under current treatments and identify factors that may predict complications.