Results for tag: "Leber Congenital Amaurosis Drug"

Leber Congenital Amaurosis (LCA) is a rare, inherited retinal disorder that causes severe vision loss in infants and young children. It is caused by mutations in one of several genes, leading to a lack of functioning photoreceptors in the retina. Treatment for LCA is limited to gene therapy, which is designed to replace the defective gene with a functioning one. The LCA drug market is a relatively small but growing segment of the optical disorders drugs market. It is driven by the increasing prevalence of LCA and the need for effective treatments. The market is expected to benefit from the development of new gene therapies and the increasing availability of clinical trials. The LCA drug market is dominated by a few large pharmaceutical companies, such as Spark Therapeutics, GenSight Biologics, and Applied Genetic Technologies Corporation. These companies are actively developing gene therapies for LCA and are expected to be the major players in the market. Other companies, such as Nightstar Therapeutics and MeiraGTx, are also developing treatments for LCA. Show Less Read more