Alglucosidase alfa

Alglucosidase alfa is a medication used to treat Pompe disease, a rare genetic disorder where a specific enzyme, acid alpha-glucosidase, is deficient. This enzyme is responsible for breaking down glycogen, a stored form of sugar, in the body's cells. Without enough enzyme, glycogen builds up, especially in muscles, causing weakness and muscle problems. Alglucosidase alfa is a man-made version of this enzyme, administered through intravenous infusions. It helps reduce glycogen buildup, improve muscle function, and manage symptoms, offering a targeted approach to addressing the underlying cause of Pompe disease.