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|Abstract:||Disclosed herein are analogs of tetrahydrobiopterin, compositions containing the same, and methods of treating an individual suffering from a condition responsive to tetrahydrobiopterin by administration of the analog. These analogs are contemplated for use wherever tetrahydrobiopterin is currently used to treat conditions responsive to tetrahydrobiopterin therapies.|
|Inventor(s):||Kakkis; Emil D. (Novato, CA)|
|Assignee:||BioMarin Pharmaceutical, Inc. (Novato, CA)|
|Filing Date:||Oct 12, 2009|
|Claims:||1. A method of treating a BH4-responsive condition or disorder, comprising administering to a subject a compound having a formula ##STR00034## or a tautomer or a pharmaceutically acceptable salt thereof, or a pharmaceutical composition comprising 2'-O-Val-BH4 or a tautomer or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable excipient, diluent or carrier; wherein the BH4-responsive condition or disorder is autism or hypertension. |
2. The method of claim 1, wherein the 2'-O-Val-BH4, or a tautomer or a pharmaceutically acceptable salt thereof, is administered in a daily dose of about 0.1 mg/kg to about 50 mg/kg in terms of BH4-equivalent amount.
3. The method of claim 1, wherein the compound or pharmaceutical composition is administered daily in a single dose, in two doses, or in more than two doses.
4. The method of claim 1, wherein the compound or pharmaceutical composition is administered orally.
5. The method of claim 1, wherein the 2'-O-Val-BH4 has a formula ##STR00035## or a tautomer or a pharmaceutically acceptable salt thereof.
6. The method of claim 1, wherein the 2'-O-Val-BH4 is a hydrochloride salt of 2'-O-Val-BH4.
7. The method of claim 1, wherein the hypertension is recalcitrant or uncontrolled hypertension, pulmonary arterial hypertension, idiopathic pulmonary hypertension, or pulmonary hypertension in the newborn (PPHN).
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