Breakthrough drugs for cystic fibrosis unveiled
Scientists have developed a treatment that offers relief and hope to people with a disease that makes every breath a struggle. The Food and Drug Administration last week approved Trikafta to treat cystic fibrosis, and doctors shared more details about the three-drug combination in journal articles and at a conference this week.
Have they found a cure for the disease? Cystic fibrosis results from a mutation in a gene that creates a protein responsible for helping manage secretions. Patients suffer from excess mucus in their lungs and other organs and often die young because of damage the disease causes. The new treatment helps the protein work properly in 90 percent of the forms of cystic fibrosis in the United States. Doctors hope to transform the course of the disease from a life-limiting illness to a manageable chronic condition such as diabetes.
Dig deeper: Read a first-hand account of the quest to cure cystic fibrosis from Francis Collins, who helped identify the gene that causes the disease in 1989 and now serves as the director of the National Institutes of Health.
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