Ariceta G, et al. - Researchers report the pathogenesis of cystinosis as well as the beneficial impacts of sustained cysteamine therapy over long-term. They found that although there is a lack of complete understanding of the exact mechanism of cystinosis but the proposed mechanism consists of accumulation and crystallization of cystine within the lysosome as a result of the lack of functional cystinosin, which subsequently leads to apoptosis and tissue damage in all organ systems. Multisystem morbidity and mortality can be seen in cases with inadequately treated cystinosis. The benefits of long-term cysteamine treatment in patients with early treatment initiation and good compliance to therapy includes delayed progression to end-stage renal disease (ESRD), remarakble improvement in growth, attenuation in the frequency and severity of extrarenal complications, and extended life expectancy. In these patients, end-organ damage can essentially be prevented and the overall prognosis can be improved with early diagnosis of cystinosis and adequate life-long treatment with cysteamine.