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.
本发明的特点是组合物、方法和试剂盒含有(i)一种或多种可在体内转化为半
胱胺的半
胱胺前体化合物,以及(ii)可选的增强这种转化的制剂,配制成一系列半
胱胺的药代动力学特征,可根据不同的患者和疾病量身定制。本发明的另一个特点是,在治疗胱
氨酸沉着症和其他半
胱胺敏感性疾病时,治疗物质的给药方式各不相同。特别是将活性成分与
药用辅料相结合的制剂,可使半
胱胺的血浆浓度保持稳定。