Results for tag: "Sandhoff Disease Drug"

Sandhoff Disease is a rare, inherited disorder that affects the central nervous system. It is caused by a deficiency of the enzyme hexosaminidase A, which is responsible for breaking down certain lipids in the body. As a result, these lipids accumulate in the brain and other organs, leading to progressive neurological damage. The Sandhoff Disease Drug market is a small but growing segment of the Central Nervous System Drugs market. Treatment options for Sandhoff Disease are limited, and currently there is no cure. However, there are several drugs available that can help to slow the progression of the disease and improve quality of life. These drugs include enzyme replacement therapy, gene therapy, and small molecule inhibitors. Companies in the Sandhoff Disease Drug market include Sanofi, Genzyme, and Shire. Sanofi is developing an enzyme replacement therapy for the treatment of Sandhoff Disease, while Genzyme is developing a gene therapy. Shire is developing a small molecule inhibitor for the treatment of the disease. Show Less Read more