Cystic Fibrosis is an incurable hereditary disorder that causes the body to produce an abnormally thick, sticky mucus that clogs the pancreas and the lungs, leading to problems with breathing and digestion, infection, and ultimately, death. Three decades ago most babies born with cystic fibrosis died in early childhood, but advances in diagnosing and treating the disease have significantly improved its outcomes. Today more than 60 percent of babies born with cystic fibrosis reach adulthood, and further advances, particularly in the field of gene therapy, may produce even better treatments in the coming years.
Cystic fibrosis is caused by a defect in the gene responsible for developing cystic fibrosis transmembrane conductance regulator (CFTR). A protein controls the flow of chloride ions into and out of certain cells.…
- American Medical Association
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