Cystinosis is a rare, autosomal recessive disease with cystine deposits in different tissues. First signs come from kidneys and eyes, but during progression of the disease other organs can also be affected. Previously, patients with cystinosis had a very poor prognosis, but it is now considerably improved due to new methods of treatment. The purpose of this paper is to give a brief review of the disease and discuss the significant improvement of the prognosis, which has been achieved by specific medical treatment with cystine-depleting agents, and, if needed, by kidney transplantation.