Pompe's disease is a lysosomal storage disorder caused by a genetic deficiency of GAA. The disease is associated with severe and progressive physical disability and premature death, and patients ...

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).

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 ...