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US 2026/0083829 A1Published application
High Concentration Alpha-Glucosidase Compositions For The Treatment Of Pompe Disease
AssigneeAmicus Therapeutics, Inc.
InventorsKenneth Valenzano, John Crowley, Richie Khanna, John Flanagan
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Abstract
The present application provides for compositions comprising high concentrations of acid α-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid α-glucosidase enzyme formulation.
Claims10
-20. (canceled)
A method of treating Pompe disease in a subject comprising administering
- (i) a first composition comprising n-butyl-deoxynojirimycin, and
- (ii) a second composition comprising acid α-glucosidase,
- wherein the acid α-glucosidase is formulated at a concentration of about 15 mg/mL in the second composition,
- wherein the acid α-glucosidase is administered by intravenous infusion at a dose of about 20 mg/kg once per two weeks and the n-butyl-deoxynojirimycin is administered orally, and
- wherein the n-butyl-deoxynojirimycin is administered prior to each administration of the acid α-glucosidase.
2 more elements
Dependencies are inferred from the claim text.