In use as therapy for Pompe disease, a recombinant human GAA produced in Chinese hamster ovary (CHO) cells is available. 10 Despite its efficacy, replacement therapy with this enzyme has some ...

CONCORD, Mass., Feb. 5, 2018 /PRNewswire/ -- Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of ...

Enzyme-replacement therapy with recombinant human GAA (rhGAA) has been a standard treatment for Pompe's disease since it was approved in 2006, they noted.

Both CRIM-positive and CRIM-negative patients with infantile-onset Pompe’s disease who were receiving enzyme-replacement therapy were reported to have anti-GAA IgG. 30-32 A negative immune ...

Pompe disease is caused by a deficiency in the lysosomal enzyme acid α-glucosidase (GAA) that leads to accumulation of glycogen in the lysosomes, mainly seen in skeletal and cardiac muscles.

Pompe is caused by mutations to the gene that codes for acid alpha glucosidase (GAA), an enzyme needed for breaking down glycogen in muscles. As a result, glycogen, a form of glucose, accumulates ...

- Award Recognizes Approval of Amicus Therapeutics’ Two-component Therapy for the Treatment of Late-onset Pompe Disease -PRINCETON, N.J., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics ...

The approval was based on data from the double-blind, active-controlled, phase 3 PROPEL trial (ClinicalTrials.gov Identifier: NCT03729362), which included 123 adults with late-onset Pompe disease ...