Cystinosis is a rare disease that usually strikes children before they are two years old and can lead to end stage kidney failure before their tenth birthday. Current treatments are limited, which is why the CIRM Board today approved $5.2 million for research that holds the possibility of a safe, effective, one-time life-long treatment.
Cystinosis is caused by a genetic mutation that allows an amino acid, cysteine, to build up in and damage the kidneys, eyes, liver, muscles, pancreas and brain of children and adults.
