Results for tag: "X Linked Severe Combined Immunodeficiency Drug"

X Linked Severe Combined Immunodeficiency (XSCID) is a rare genetic disorder that affects the immune system. It is caused by a mutation in the gene that produces the enzyme adenosine deaminase (ADA). Without ADA, the body cannot produce enough T-cells, which are essential for fighting off infections. As a result, people with XSCID are highly susceptible to infections and other illnesses. The XSCID drug market is focused on treatments that can restore the body’s ability to produce ADA. These treatments include gene therapy, enzyme replacement therapy, and stem cell transplantation. Gene therapy involves introducing a healthy copy of the ADA gene into the patient’s cells, while enzyme replacement therapy involves administering ADA directly to the patient. Stem cell transplantation is a more complex procedure, but it can be used to replace the patient’s defective immune system with a healthy one. The XSCID drug market is relatively small, but it is growing as more treatments become available. Companies in the market include bluebird bio, AveXis, and Spark Therapeutics. These companies are developing treatments that can help restore the body’s ability to produce ADA and improve the quality of life for people with XSCID. Show Less Read more