The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Site Search
I grew up on a farm in the countryside in Sweden. My younger brother also has cystic fibrosis, so money was always a little tight in my family, due to the costs of treatments. My classmates would often brag about places they had been on their vacation, England, Stockholm, Greece, and I was very envious.
Planning a last-minute vacation before the end of the summer? We’ve got you covered! All week long we’ll be sharing tips, advice and personal stories on traveling with CF.
CF Foundation's $1 Million Investment Helped Drug Known as Cayston® Become a Reality
New Effort Will Search for Potential Therapies Targeting the Most Common Mutation of Cystic Fibrosis
Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments
This medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually benefit more than 90 percent of people with CF.
After starting on Trikafta®, I finally became able to travel without getting an immediate exacerbation when I returned home. Now, I am relishing the opportunity to travel abroad, while taking every precaution necessary to protect myself from the pandemic.