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.
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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
I struggled emotionally after learning that after negative carrier and newborn screens, my son had cystic fibrosis. I’m now advocating for people with rare mutations.
My son, who is Black, has cystic fibrosis. It seems like just a normal fact of life, but I have become frustrated with having to convince doctors that he really does have CF. I hope that one day people of color won’t have to have the same experience.
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.
The Cystic Fibrosis Foundation's successful business model was at the center of a congressional briefing in Washington, D.C., today, which focused on strategies for jump-starting drug development for rare diseases.
Corbus Pharmaceuticals reported promising results Thursday from an early stage clinical study of a potential anti-inflammatory drug for people with cystic fibrosis.
Cystic fibrosis affects people of every racial and ethnic group. Of the nearly 40,000 people living with CF in the U.S., approximately 15% are identified as racially or ethnically diverse. Research shows that people of color with CF, particularly people who are Black and Hispanic, experience unique challenges and often have negative experiences that can lead to poorer outcomes. Some of these inequities are referenced in the following data.