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Suppository formulations as a potential treatment for nephropathic cystinosis.

机译:栓剂可作为肾病性胱氨酸病的一种潜在治疗方法。

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摘要

Nephropathic cystinosis is a rare autosomal recessive disease characterised by raised lysosomal levels of cystine in the cells of all the organs. It is treated by the 6-h oral administration of the aminothiol, cysteamine, which has an offensive taste and smell. In an attempt to reduce this frequency and improve the treatment, cysteamine-containing polyethylene glycol suppositories were prepared and evaluated for dissolution and stability. The results demonstrated that cysteamine release was complete after 30 min, and that there was a uniform drug distribution within the formulations. Twelve-month stability tests highlighted a potential incompatibility among some excipients, although stability was demonstrated for the cysteamine suppositories up to 6 months. These suppositories may provide a useful alternative to the current oral therapy for cystinosis.
机译:肾病性胱氨酸病是一种罕见的常染色体隐性遗传疾病,其特征是所有器官细胞中胱氨酸的溶酶体水平升高。通过6小时口服氨基硫醇,半胱胺进行治疗,这种氨基硫醇具有令人讨厌的味道和气味。为了降低这种频率并改善治疗效果,制备了含半胱胺的聚乙二醇栓剂,并评价了其溶出度和稳定性。结果表明,半胱胺释放在30分钟后完成,并且制剂中药物分布均匀。十二个月的稳定性测试强调了某些赋形剂之间的潜在不相容性,尽管半胱胺栓剂的稳定性可长达6个月。这些栓剂可以为当前口服治疗胱氨酸病提供一种有用的替代方法。

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