Results for tag: "Canavan Disease Drug"

Canavan Disease is a rare, inherited disorder of the central nervous system caused by a deficiency of the enzyme aspartoacylase. It is characterized by progressive deterioration of the brain, leading to mental and physical disability. Treatment for Canavan Disease is limited to supportive care, and there is currently no approved drug therapy. The Canavan Disease Drug market is a small but growing segment of the Central Nervous System Drugs market. Companies in this market are focused on developing treatments for Canavan Disease, including gene therapy, enzyme replacement therapy, and small molecule drugs. These treatments are still in the early stages of development, and there is no approved drug therapy for Canavan Disease yet. Companies in the Canavan Disease Drug market include Lysogene, Bluebird Bio, and Ultragenyx Pharmaceuticals. Lysogene is developing a gene therapy for Canavan Disease, while Bluebird Bio is developing an enzyme replacement therapy. Ultragenyx Pharmaceuticals is developing a small molecule drug for Canavan Disease. Show Less Read more