METHODS OF TREATING UREA CYCLE DISORDERS

摘要

The present disclosure provides novel methods for determining an effective dosage of a PAA prodrug and for treating a UCD that incorporate body surface area and urinary PAGN concentration. The disclosure further provides novel methods for assessing compliance with PAA prodrug administration that incorporate urinary PAGN concentration, and the subject's current dosing regimen, BSA, or age. The disclosure further provides novel methods of treating a UCD in a subject in need thereof that incorporate urinary PAGN concentration, and the subject's current dosing regimen, BSA, and/or age.

主权项

1. A method of determining an effective dosage of a phenylacetic acid (PAA) prodrug for treating a urea cycle disorder in a subject in need thereof comprising:
calculating a body surface area (BSA) for the subject; and comparing the BSA to a predetermined threshold value, wherein the effective dosage is a first dosage if the BSA is at or above the predetermined threshold value or a second dosage if the BSA is below the predetermined threshold value, and wherein the second dosage is higher than the first dosage as a function of BSA.