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Evaluation of a novel drug combination treatment for nephropathic cystinosis in a new cystinotic rat model.
Cystinosis is a rare disease that results in the build-up of cystine in all cells of the body. Cystine is a protein building block and normally the excess is moved out of cells. In cystinosis the transporter for cystine does not work and cystine builds up inside the cells and forms crystals which cause damage to all organs and...