Claims for Patent: 11,202,767

Summary for Patent: 11,202,767

Title:	Methods of treating urea cycle disorders and maple syrup urine disease
Abstract:	The present invention provides methods for the treatment of urea cycle disorders and Maple Syrup Urine Disease.
Inventor(s):	Appel Leah E., Shockey Joshua R., Schelling D. Christopher
Assignee:	Acer Therapeutics Inc.
Application Number:	US17196599
Patent Claims:	2. The method of claim 1 , wherein claim 1 , upon administration to the subject claim 1 , the pharmaceutical composition is bioequivalent to compared to a sodium phenylbutyrate formulation that does not comprise a taste-mask coating.3. The method of claim 1 , wherein the pharmaceutical composition is administered in combination with a dosing vehicle.4. The method of claim 3 , wherein the dosing vehicle comprises modified food starch claim 3 , maltodextrin claim 3 , or a combination thereof.5. The method of claim 4 , wherein the modified food starch is modified corn starch.6. The method of claim 1 , wherein the therapeutically effective amount is 450-600 mg/kg/day in subjects weighing less than 20 kg or 9.9-13.0 g/m/day in larger subjects.7. The method of claim 1 , wherein the subject is on a protein restricted diet.8. The method of claim 1 , further comprising administering essential amino acids to the subject.9. The method of claim 1 , wherein the therapeutically effective amount is administered in equally divided amounts with each meal or feeding.10. The method of claim 1 , wherein the therapeutically effective amount is administered three to six times per day.11. The method of claim 1 , wherein the urea cycle disorder is caused by a mutation in N-acetylglutamate synthetase claim 1 , carbamoyl phosphate synthetase I claim 1 , ornithine transcarbamylase claim 1 , argininosuccinic acid sythetase claim 1 , argininosuccinic acid lyase claim 1 , or arginase.12. The method of claim 1 , wherein the plurality has a volume-based particle size distribution in which at least 90% of the layered particles in the plurality of layered particles are smaller than 500 μm.13. The method of claim 1 , wherein the subject is a human.14. The method of claim 1 , wherein the administration results in removal of excess ammonia from the blood of the subject.16. The method of claim 15 , wherein the pharmaceutical composition is administered in combination with a dosing vehicle.17. The method of claim 16 , wherein the dosing vehicle comprises modified food starch claim 16 , maltodextrin claim 16 , or a combination thereof.18. The method of claim 17 , wherein the modified food starch is modified corn starch.19. The method of claim 15 , wherein the therapeutically effective amount is 450-600 mg/kg/day in subjects weighing less than 20 kg or 9.9-13.0 g/m/day in larger subjects.20. The method of claim 15 , wherein the therapeutically effective amount is administered in equally divided amounts with each meal or feeding.21. The method of claim 15 , wherein the therapeutically effective amount is administered three to twelve times per day.22. The method of claim 15 , wherein the plurality has a volume-based particle size distribution in which at least 90% of the layered particles in the plurality of layered particles are smaller than 500 μm.23. The method of claim 15 , wherein the subject is a human.24. The method of claim 15 , wherein the therapeutically effective amount is sufficient to lower the plasma level of a branched chain amino acid (BCAA).25. The method of claim 24 , wherein the BCAA is leucine claim 24 , and the level is lowered to 200-500 μmol/L.26. The method of claim 24 , wherein the BCAA is isoleucine claim 24 , and the level is lowered to 100-200 μmol/L.27. The method of claim 24 , wherein the BCAA is valine claim 24 , and the level is lowered to 100-300 μmol/L.