Cysteamine therapy: a treatment for cystinosis, not a cure
Stephanie Cherqui, Stephanie Cherqui
Abstract
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.
Conflict of interest statement
DISCLOSURE
The author declared no competing interests.
Figures
Source: PubMed