Results for tag: "Pompe Disease Drug"

Pompe Disease is a rare, inherited disorder caused by a deficiency of the enzyme alpha-glucosidase. This deficiency leads to the accumulation of glycogen in the lysosomes of cells, resulting in progressive muscle weakness and respiratory failure. Endocrine and Metabolic Disorders Drugs are used to treat Pompe Disease. These drugs are designed to replace the missing enzyme, allowing the body to break down the accumulated glycogen and restore normal muscle function. The Pompe Disease Drug market is a rapidly growing segment of the Endocrine and Metabolic Disorders Drugs market. The increasing prevalence of Pompe Disease and the availability of effective treatments have driven the growth of this market. Companies in this market are focused on developing treatments that are safe and effective, as well as improving existing treatments. Some companies in the Pompe Disease Drug market include Sanofi, Pfizer, Amicus Therapeutics, and Ultragenyx Pharmaceuticals. Show Less Read more