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US 2026/0216302 A1Published application
Compositions and methods for treating late-onset pompe disease
AssigneeGenzyme Corporation
InventorsPatrick FINN, Alaa HAMED, Kristina AN HAACK, Catherine WILSON
Life of this patent
Estimating the term…
Abstract
The present application provides methods of treating late-onset Pompe disease (LOPD) using a pharmaceutical composition comprising an oligosaccharide-acid α-glucosidase (GAA) conjugate, such as avalglucosidase alfa.
Claims23
1-69. (canceled)
A method for increasing health-related quality of life of a human individual having a late-onset Pompe disease (LOPD), comprising administering to the individual a pharmaceutical composition comprising an oligosaccharide-protein conjugate and a pharmaceutically acceptable carrier,
- wherein the oligosaccharide-protein conjugate is avalglucosidase alfa,
- wherein the pharmaceutical composition is administered at a dose of about 5 mg/kg to about 40 mg/kg,
- wherein administration of the pharmaceutical composition for 49 weeks to a plurality of individuals results in one or more of the following:
- (a) a decrease in EuroQol 5 Dimensions 5 Response levels (EQ-5D-5L) Mobility score of at least about 0.3;
- (b) a decrease in EQ-5D-5L Self-Care score of at least about 0.1;
- (c) a decrease in EQ-5D-5L Usual Activities score of at least about 0.1;
- (d) a decrease in EQ-5D-5L Pain/Discomfort score of at least about 0.1;
- (e) a decrease in EQ-5D-5L Anxiety/Depression score of at least about 0.1;
- (f) an increase in EuroQol 5 Dimensions Visual Analogue Scale (EQ-VAS) score of at least about 4.3;
- (g) an increase in Rasch-built Pompe-specific Activity (R-Pact) score of at least about 1.3;
- (h) an increase in Patient Global Impression of Change (PGIC) Daily Activities score of at least 4.6;
- (i) an increase in PGIC Disease-Related Symptoms score of at least 4.4;
- (j) an increase in PGIC Ability to Breath score of at least 4.8;
- and
- (k) an increase in PGIC Mobility score of at least 4.4.
12 more elements
Dependencies are inferred from the claim text.