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
Like many of my millennial compatriots, I was booted from my parents' health plan when I turned 26. While my friends were shrugging health insurance off as just another growing pain of their 20s, I was panicking.
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.
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.
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