Cystinosis Treatment; Depends On the Age, Type, and the Degree of Disease Severity
In the past, cystinosis treatment was limited to treat metabolic acidosis and replace electrolytes lost in the urine. Today, the wide availability of an effective drug, kidney transplantation, and cysteamine, has dramatically improved the treatment and management of cystinosis. Cystinosis is the most common hereditary cause of renal Fanconi syndrome in children; characterized by accumulation of the amino acid cystine within cells. Excess cystine damages cells and forms crystals that can build up and cause problems in many organs and tissues. Kidney transplantation is a safe and effective treatment for the kidney failure of individuals with cystinosis. Nephropathic (infantile) cystinosis is the most common and severe form of the cystinosis. Nephropathic cystinosis usually appears in infants and children at a young age. It is a life-long condition, but available treatments, such as kidney transplantation and cysteamine therapy, have allowed people with the disease to live longer. Int