COMPASSIONATE USE OF CYSTEAMINE IN NEPHROPATHIC CYSTENOSIS

$0M01FY2000RRNIH

Mayo Clinic Coll Of Medicine, Rochester, Rochester MN

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Abstract

Nephropathic cystinosis is an inherited disease with multiple clinical manifestations including Fanconi's syndrome and progressive kidney failure. Within the past decade, cysteamine and phosphocysteamine were developed as orphan drugs. These compounds have now been found to be effective in reducing the rate of progression of cystinosis. However, multiple daily doses of medication and regular monitoring of white blood cell cystine levels are essential to optimal outcomes.

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