METHODS OF TREATING UREA CYCLE DISORDERS
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.
1 - 20 . (canceled)
21 . A method for treating a urea cycle disorder (UCD) in a subject in need thereof who is less than two years of age comprising administering to the subject an therapeutically-effective dosage of glyceryl tri-[4-phenylbutyrate].
22 . The method of claim 23 , wherein the glyceryl tri-[4-phenylbutyrate] is administered orally.
23 . The method of claim 21 , wherein the therapeutically-effective dosage is 5 to 12.4 g/m 2 /day.
24 . The method of claim 23 , wherein the therapeutically-effective dosage is of about 6.25 to 9.9 g/m 2 /day.
25 . The method of claim 24 , wherein the therapeutically-effective dosage is at or about 7 to 9.5 g/m 2 /day, 7.5 to 9 g/m 2 /day, 8.0 to 8.5 g/m 2 /day, or 8.3 to 8.5 g/m 2 /day.
26 . The method of claim 25 , wherein the therapeutically-effective dosage is at or about 8.02 of 8.35 g/m 2 /day.
27 . The method of claim 21 , wherein the subject has not previously received a phenylbutyrate-based drug.