Cysteamine therapy delays the progression of nephropathic cystinosis in late adolescents and adults.

  title={Cysteamine therapy delays the progression of nephropathic cystinosis in late adolescents and adults.},
  author={Albane Brodin-Sartorius and Marie-Jos{\`e}phe T{\^e}te and Patrick Niaudet and Corinne Antignac and Genevi{\`e}ve Guest and Chris Ottolenghi and Marina Charbit and Dominique Moyse and C. Legendre and Philippe Lesavre and Pierre Cochat and Aude Servais},
  journal={Kidney international},
  volume={81 2},
Nephropathic cystinosis is a multisystem autosomal recessive disease caused by cystine accumulation, which is usually treated by oral cysteamine. In order to determine long-term effects of this therapy, we enrolled 86 adult patients (mean age 26.7 years) diagnosed with nephropathic cystinosis, 75 of whom received cysteamine. Therapy was initiated at a mean age of 9.9 years with a mean duration of 17.4 years. By last follow-up, 78 patients had end-stage renal disease (mean age 11.1 years), 62… CONTINUE READING
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