Alpha-1 antitrypsin deficiency is an inherited genetic disorder that affects mainly the lungs. It limits even mild activity and can be fatal. Intensive research continues in order to find even better treatments.
Disease: Alpha-1 antitrypsin deficiency
Last update: June 2006
Intro
FAQ: What does the future hold?
Researchers are investigating new anti-inflammatory medicines. Research has also discovered that the cause of the disease is due to a faulty enzyme controlled by a single gene on chromosome 14. Gene therapy - to replace the faulty gene - remains the ultimate goal to ameliorate or cure the disease.