Cysteamine for Cystinosis

Cysteamine bitartrate (Cystagon - Mylan), an orphan drug, has been approved by the US Food and Drug Administration for oral treatment of nephropathic cystinosis, an autosomal recessive metabolic disorder that affects only a few hundred people in the USA (WA Gahl et al, in The Metabolic Basis of Inherited Disease, 7th ed., CR Scriver et al, eds, New York:McGraw-Hill, 1995, p 3763)....

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