Amicus Therapeutics, Inc.

John Crowley

John R Flanagan

Richie Khanna

Kenneth Valenzano

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.

High concentration alpha-glucosidase compositions for the treatment of Pompe disease

The present application provides for compositions comprising high concentrations of acid a-glucosidase in combination with an active site-specific chaperone for the acid &#x3b1;-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 &#x3b1;-glucosidase enzyme formulation.

High concentration alpha-glucosidase compositions for the treatment of pompe disease

The present application provides for compositions comprising high concentrations of acid &#x3b1;-glucosidase in combination with an active site-specific chaperone for the acid &#x3b1;-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 &#x3b1;-glucosidase enzyme formulation.

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Princeton, NJ, United States of America

John Crowley