Does enhanced vitamin D activity in bone heal the skeleton in disorders of FGF23 excess?

骨骼中维生素 D 活性的增强能否治愈 FGF23 过量疾病中的骨骼？

• 批准号: nhmrc : GNT1130338
• 负责人: A/Pr Paul Anderson
• 金额: $ 85.59万
• 依托单位: University of South Australia
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2017
• 资助国家: 澳大利亚
• 起止时间: 2017-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/does-enhanced-vitamin-fgf23-excess/1347807
• 关键词: Does; enhanced; vitamin; activity; bone

X-linked hypophosphatemia (XLH) is a genetic disorder which results in phosphate wasting and rickets. This severe disorder has no effective treatment. We have compelling new evidence that the rickets in XLH is not primarily a disorder of low blood phosphate, but rather specific issue of low cellular levels and activity of vitamin D (1,25D) within bone. This proposal is designed to specifically demonstrate this new concept and outline a new paradigm for a new XLH treatment.

X连锁低磷血症（XLH）是一种遗传性疾病，可导致磷酸盐消耗和佝偻病。这种严重的疾病没有有效的治疗方法。我们有令人信服的新证据表明，XLH中的佝偻病主要不是低血磷的疾病，而是骨内维生素D（1，25D）的细胞水平和活性低的具体问题。该提案旨在具体展示这一新概念，并概述新XLH治疗的新范例。