Is Trikafta a cure?

Summary. Trikafta® is a highly effective therapy for the underlying cause of cystic fibrosis. It is approved for use in people with CF ages 6 and older who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data. It is not a cure for CF.

How effective is Trikafta?

Trikafta is the first approved treatment that is effective for cystic fibrosis patients 12 years and older with at least one F508del mutation, which affects 90% of the population with cystic fibrosis or roughly 27,000 people in the United States.

What is the mechanism of ivacaftor?

Mechanism of Action Ivacaftor facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the G551D-CFTR protein. Cystic fibrosis is caused by mutations in a gene that encodes for the CFTR protein that regulates ion (such as chloride) and water transport in the body.

How old is the longest living person with cystic fibrosis?

Thanks to advances in DNA testing, doctors are identifying more and more people with CF for the first time well into their 50s, 60s, and 70s. The oldest person diagnosed with CF for the first time in the U.S. was 82, in Ireland was 76, and in the United Kingdom was 79.

Will Trikafta increase life expectancy?

Use of Trikafta could mean an increase in the life expectancy of cystic fibrosis patients, and for young children who begin the treatment early, “They might never have to experience the symptoms of cystic fibrosis in the same way prior to Trikafta,” Quinn said.

Is ivacaftor a gene therapy?

Ivacaftor (Vx-770; Kalydeco, Vertex Pharmaceuticals, Cambridge, MA) is a new and novel gene-based therapy for the treatment of CF.

What type of drug is ivacaftor?

Ivacaftor is in a class of medications called cystic fibrosis transmembrane conductance regulator (CFTR) potentiators. It works by improving the function of a protein in the body to decrease the build-up of thick mucus in the lungs and improving other symptoms of cystic fibrosis.

Can you live a long life with cystic fibrosis?

While there is no cure yet for cystic fibrosis (CF), people with CF are living longer, healthier lives than ever before. In fact, babies born with CF today are expected to live into their mid-40s and beyond. Life expectancy has improved so dramatically that there are now more adults with cystic fibrosis than children.

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