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

x连锁低磷血症（XLH）是一种遗传性疾病，导致磷酸盐浪费和佝偻病。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酸盐的疾病，而是骨内维生素D （1,25D）细胞水平和活性低的特定问题。本提案旨在具体展示这一新概念，并概述新的XLH治疗的新范例。

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.