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Compositions for controlled release of cysteamine and systemic treatment of cysteamine sensitive disorders
Compositions for controlled release of cysteamine and systemic treatment of cysteamine sensitive disorders
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机译:用于控制半胱胺释放和全身治疗半胱胺敏感性疾病的组合物
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摘要
The invention features compositions, methods, and kits containing (i) one or more cysteamine precursor compounds convertible to cysteamine in vivo, and (ii) optionally agents to enhance that conversion, formulated to produce a spectrum of pharmacokinetic profiles of cysteamine that can be tailored to individual patients and diseases. The invention also features varying modes of administration of the therapeutic substances in the treatment of cystinosis and other cysteamine sensitive disorders. In particular, formulations combining active ingredient(s) with pharmaceutical excipients that permit sustained cysteamine plasma concentrations are featured.
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