Cysteamine therapy in nephropathic cystinosis

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Cysteamine therapy is most effective in nephropathic  cystinosis when started early in life. However, a recent study by Brodin-Sartorius points out that there are still clinical improvements seen if this therapy is initiated in older children.  They found that initiating therapy <5 years of age significantly improved renal, diabetes, and thyroid outcome. However in chldren starting therapy after 5 years of age, there was still significantly delayed development of diabetes and hypothyroidim. (Brodin-Sartorius et al. Kidney Int. 2011 Sep 7 [epub].)

Hilary Vernon MD PhD

http://dx.doi.org/10.1036/ommbid.233

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