Inhibiting FGF23 for bone defects in β-thalassemia

Inhibiting signaling downstream of FGF23, a hormone that inhibits bone mineralization and parathyroid hormone production, could help treat bone defects in β-thalassemia patients by normalizing the bone marrow stem cell niche.

In plasma samples from β-thalassemia patients with bone defects, FGF23 levels were higher than in samples from healthy controls, and higher levels of FGF23 were positively correlated with severity of bone degradation and negatively correlated with hemoglobin concentration. ...