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.

Purpose: Infantile glycogen storage disease type II (GSD-II) is a fatal genetic muscle disorder caused by deficiency of acid α-glucosidase (GAA). The purpose of this study was to investigate the ...

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

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

POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who ...

Get detailed information on Voglibose, including pronunciation, uses, dosage guidelines, indications, and instructions on how and when to take it and when to avoid it. The updated prescription ...

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: ...