PEGylated derivatives of cystamine as en
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Ziad Omran; Graeme Kay; Alberto Di Salvo; Rachel M. Knott; Donald Cairns
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Article
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2011
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Elsevier Science
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English
β 285 KB
The genetic disease, nephropathic cystinosis is characterized by lysosomal accumulation of the amino acid cystine. Crystallization of cystine in affected organs, if untreated, results in mortality of the affected individuals by their middle to late teens. The only approved treatment for cystinosis i