Dose response of bone-targeted enzyme replacement for murine hypophosphatasia

► We find a positive correlation between ENB-0040 dose and prevention of mineralization defects in a murine model of hypophosphatasia. ► We find a positive relationship between ENB-0040 dose and survival. ► Urinary pyrophosphate is not a good follow up marker for evaluating response when using bone-targeted TNSALP treatment. ► These studies help set the therapeutic range of ENB-0040 to be chosen for clinical trials.