A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis
Abstract Background Nephropathic cystinosis is a rare and severe metabolic disease leading to an accumulation of cystine in lysosomes which especially harms kidney function. A lifelong therapy with the aminothiol cysteamine can delay the development of end-stage renal disease and the necessity of ki...
|Main Authors:||, , , , , , ,|
|Series:||Orphanet Journal of Rare Diseases|