AMINOTHIOL THERAPY IN NEPHROPATHIC CYSTINOSIS

氨基硫醇治疗肾病性胱氨酸贮积症

• 批准号: 5217669
• 负责人: JESS G THOENE
• 金额: --
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/5217669
• 关键词: clinical research; cysteamine; drug metabolism; drug screening /evaluation; human subject; pharmacokinetics

The investigational form of cysteamine (hydrochloride) will be required only for those patients unable to tolerate the capsule formulation. Some infants may require an aqueous solution. This form will be continued in IND status by Dr. Thoene who is the sponsor. Patients receiving it will be studied to compare white blood cell cystine depletion between this form and the FDA-approved form of the drug.

将需要半胱胺（盐酸）的研究形式