Methods of treating urea cycle disorders
AssigneeHorizon Therapeutics, LLC
InventorsBruce SCHARSCHMIDT, Masoud MOKHTARANI
Life of this patent
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Abstract
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.
Claims8
1-20. (canceled)
A method of evaluating compliance with a phenylacetic acid (PAA) prodrug treatment regimen in a subject with a urea cycle disorder (UCD) being treated with a PAA prodrug, wherein the PAA prodrug is glyceryl tri-[4-phenylbutyrate], said method comprising:
- classifying the subject into a dosage group based on the dosage of a PAA prodrug the subject is currently receiving;
- determining a urinary phenylacetylglutamine (PAGN) level for the subject;
- and
- comparing the urinary PAGN level to a predetermined threshold urinary PAGN level,
- wherein a urinary PAGN level below the predetermined threshold urinary PAGN level indicates that the subject is non-compliant with the PAA prodrug treatment regimen.
Dependencies are inferred from the claim text.