Cysteamine therapy: a treatment for cystinosis, not a cure

Kidney Int. 2012 Jan;81(2):127-9. doi: 10.1038/ki.2011.301.


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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Comment

MeSH terms

  • Animals
  • Bone Marrow Transplantation*
  • Cystinosis / surgery*
  • Hematopoietic Stem Cell Transplantation*
  • Kidney / pathology*
  • Kidney Diseases / prevention & control*