Pompe disease is characterized by a deficiency of acid α-glucosidase (GAA). The GAA enzyme degrades lysosomal glycogen, and insufficient GAA activity causes glycogen to accumulate in various tissues.

11.3. The Advisory Committee recommended that avalglucosidase alfa for the treatment of late onset Pompe disease be funded with a medium priority.

Diabetes is a metabolic disease caused by insulin deficiency that leads to high blood sugar levels and several associated complications if left untreated.

Q3 2024 Total Revenue of $141.5M, a 37% Increase Year-over-Year Galafold®Q3 Revenue of $120.4M, up 20% Year-over-Year PRINCETON, N.J., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: ...