The U.S. Food and Drug Administration (FDA) approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 6 to 11, who have two copies of the F508del mutation.
Site Search
The U.S. Food and Drug Administration (FDA) has decided not to approve the use of ivacaftor (Kalydeco®) to treat people with cystic fibrosis ages 2 and older who have one of 23 rare CF mutations, known as “residual function” mutations.
As some people's pandemic fears subside and businesses start to reopen in parts of the U.S., people with cystic fibrosis like me are maintaining their social distance. We still need to be extra cautious because of the threat COVID-19 poses to people with an underlying condition.
Clinical trials that test potential drugs and therapies in people with cystic fibrosis are a major part of CF research. They take place at Cystic Fibrosis Foundation-accredited care centers all over the United States and enroll people with CF of all ages.
When you are deciding whether to join a clinical trial, you should consider logistics.
As a person with cystic fibrosis, I struggled all my life to gain weight, despite eating a high-calorie, high-fat diet. Once I started Trikafta® and my weight increased to normal levels, I had to learn a new, healthier way to eat.
After years of hospitalizations and low lung function, I am now feeling healthy on Trikafta® -- so much so that I finally feel like I can reset the clock and start living my life.
If not for my mother and my wife, I would not have survived this arduous, 50-year journey with cystic fibrosis. But now I have another ally: Trikafta® is having a substantial impact on my health.
It took me 38 years to find a fitness program to stick to. And since I started TrikaftaTM, I am even running for the first time in my life -- and almost enjoying it!
I'm a big proponent of participating in clinical research, but COVID-19 made me think twice about participating in a clinical trial that required six hospital visits. However, a screening visit reassured me that the researchers were taking all the necessary precautions.