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Suppository formulations as a potential treatment for nephropathic cystinosis

✍ Scribed by Barbara Buchan; Graeme Kay; Kerr H. Matthews; Donald Cairns


Book ID
112134655
Publisher
John Wiley and Sons
Year
2012
Tongue
English
Weight
283 KB
Volume
101
Category
Article
ISSN
0022-3549

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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