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 ...

About Late-Onset Pompe Disease Late-onset Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA).

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.

The biotechnology company explained that Pombiliti is a recombinant human GAA enzyme naturally expressed with high levels of bis-M6P, designed for increased uptake into muscle cells, whereas ...

About Late-Onset Pompe Disease Late-onset Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA).

Pombiliti is a recombinant human GAA enzyme designed for increased uptake into muscle cells, while Opfolda functions as an enzyme stabilizer in the blood.

Pombiliti is a recombinant human GAA enzyme designed for increased uptake into muscle cells, while Opfolda functions as an enzyme stabilizer in the blood. The approval was based on clinical data from ...

About Late-Onset Pompe Disease Late-onset Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to ...