Pharmaceutical compositions comprising sepiapterin and uses thereof
AssigneePTC Therapeutics MP, Inc.
InventorDaniel E. Levy
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Abstract
The present invention features pharmaceutical compositions including sepiapterin, or a pharmaceutically acceptable salt and/or co-crystal thereof, and methods for the treatment of tetrahydrobiopterin-related disorders (e.g., tetrahydrobiopterin deficiency or phenylketonuria) with such compositions.
Claims19
A method of treating a tetrahydrobiopterin-related disorder selected from tetrahydrobiopterin deficiency and phenylketonuria in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition comprising an effective amount of sepiapterin and a pharmaceutically acceptable carrier,
- wherein the relative amount of antioxidant to sepiapterin is between 0 and 20% wt/wt;
- wherein the antioxidant, when present, is ascorbic acid, an ester of ascorbic acid, or a salt of ascorbic acid;
- and
- wherein from about 2.5 mg sepiapterin per kilogram of body weight of the subject (mg/kg) to about 150 mg/kg is administered.
Dependencies are inferred from the claim text.