Results for tag: "Mucopolysaccharidosis Drug"

Mucopolysaccharidosis (MPS) is a group of rare, inherited metabolic disorders caused by the deficiency of lysosomal enzymes. These enzymes are responsible for breaking down complex sugars called glycosaminoglycans (GAGs). Without these enzymes, GAGs accumulate in the body, leading to a wide range of symptoms, including skeletal deformities, organ enlargement, and cognitive impairment. Treatment for MPS is focused on managing symptoms and preventing further progression of the disease. Immune disorders drugs are used to treat MPS, including enzyme replacement therapy (ERT), substrate reduction therapy (SRT), and gene therapy. ERT involves the administration of a recombinant enzyme to replace the missing enzyme, while SRT works by reducing the production of GAGs. Gene therapy is a newer approach that involves the introduction of a healthy gene into the patient's cells to replace the defective gene. Companies in the MPS drug market include Sanofi, Shire, Pfizer, and Ultragenyx Pharmaceuticals. Sanofi is a French pharmaceutical company that produces Aldurazyme, an ERT drug for MPS I. Shire is a biopharmaceutical company that produces Elaprase, an ERT drug for MPS II. Pfizer is an American pharmaceutical company that produces Vimizim, an ERT drug for MPS IV. Ultragenyx Pharmaceuticals is a biopharmaceutical company that produces Mep Show Less Read more