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Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits a person’s ability to breathe over time. The average life expectancy of a person with cystic fibrosis is approximately 37 years. Findings from a recent clinical trial indicate that a new drug may help manage the symptoms of the disease and extend their lifespan.
The phase 3, randomized, double-blind, placebo-controlled trial involved more than 400 people aged 12 years and older who have cystic fibrosis. Participants were randomized to receive either the new drug, commonly known as Trikafta, or a placebo for 24 weeks.
At the end of the 24-week period, the participants' lung capacity was measured. Those who received the Trikafta scored 14.3 points higher on lung function tests, were 63 percent less likely to have worsening of symptoms, and had better quality of life compared to those given a placebo. Trikafta was considered safe and elicited few, mild side effects.
The drug works by targeting the underlying genetic mutation that causes the most common form of the disease.