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Cystic Fibrosis Patients Celebrate FDA Approval for "Changing Life" of New Treatment



The recent FDA approval of three-drug combination therapy for patients with cystic fibrosis has been reported by both patients and physicians as "life-changing" news. Trikafta, manufactured by Vertex Pharmaceuticals Inc., targets the defective protein responsible for the disease that causes the thick mucus to accumulate in the lungs and other organs.

This is the first triple combination therapy available to treat at least one F508del mutation in the gene for CFM, which is the most common in patients. Among those who qualify for Trikafta is Gunnar Esiason, the son of former NFL quarterback Boomer Esiason, who began treatment in a clinical trial last April. My life changed on April 9, 201

8, when I took my first dose of Triple Combo in a Phase III clinical trial, ”Gunnar Esiason tweeted on October 21, the day the FDA announced its decision. "A triple combo named after its various components is a medicine designed to treat the underlying cause of cystic fibrosis. There was a 50/50 shot that I would take the active drug or placebo, but I knew I had the real thing because it worked for hours. Everything has changed since then. "

Gunnar Esiasson said the health agency's decision would put the treatment in the hands of more patients and that he" couldn't wait to see how people with CF work to change the world now that almost our entire community will has access to this life-changing medicine. "

The treatment, although not mild, is for patients who have at least one copy of the F508del mutation and include taking several pills every day. According to Gunnar Esiason, this has helped him "continue his life largely independent of the severe symptoms that come with cystic fibrosis." studies of patients with two copies of the F508del mutation had a 10 percent increase in lung function, and people with a single copy had a more than 14 percent increase compared to placebo.

"The medicine gave me the life that was stolen from me while I was crawling for the disease in the final stages," he tweeted.

The Cystic Fibrosis Foundation also called FDA approval a "huge breakthrough" for patients with the disease. people live with cystic fibrosis in the US, with an estimated 1,000 new cases being diagnosed every year, according to the MB Foundation.More than half of the patient population is 18 years of age or older and it is estimated that the drug can be made available to more than 90 percent of patients.

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"As we note today, we will not lose sight of the many people in our community who are still waiting for a breakthrough to cure their mutations," said Michael P. Boyle , PhD, Senior Vice President, CF Therapy Foundation Development Therapist "We will not act until every person with CF has treated the underlying cause of their illness and one day has a cure."


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