Cysteamine therapy: a treatment for cystinosis, not a cure.
Cystinosis as a clinical entity is a progressive dysfunction of multiple organs caused by the accumulation of cystine in the tissues, leading, for example, to end-stage renal failure, diabetes, hypothyroidism, myopathy, and central nervous system deterioration. Brodin-Sartorius and colleagues present a long-term study on the impact of cysteamine therapy on these complications. The data show that cysteamine improves the outcome and complications of cystinosis but does not prevent them.
Stephanie
S
Cherqui
D000818:Animals; D016026:Bone Marrow Transplantation; D003554:Cystinosis / Q000601:surgery*; D018380:Hematopoietic Stem Cell Transplantation; D007668:Kidney / Q000473:pathology*; D007674:Kidney Diseases / Q000517:prevention & control*