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A Possible Cure?

In 1985, the specific gene that causes cystic fibrosis was identified. That same year, a screening test for newborns was introduced. Laboratory tests of gene therapy have been successful. Small group tests of a genetically engineered enzyme have also been successful. The enzyme, administered in aerosol form, thins out the mucus in the patient’s lungs, making it easier to clear by coughing. Testing on this treatment continues, and the U.S. Food and Drug Administration has approved it. Life expectancy for cystic fibrosis patients continues to increase. With all of the investigations underway in gene therapy, improved drugs, and transplant experience, the future of cystic fibrosis patients appears brighter than ever.

 

2024 American Association for Respiratory Care