Cipaglucosidase alfa (ATB200) is a novel recombinant human acid alpha-glucosidase (GAA) investigated for the treatment of patients with Pompe disease, a rare inherited metabolic disorder characterized by a deficiency in GAA.[A232955] Other types of enzyme rep…

Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be …