The Cystic Fibrosis Foundation is aggressively pursuing potential treatments for people with CF who have nonsense and rare mutations who will not benefit from drugs known as modulators, which correct the malfunctioning cystic fibrosis transmembrane conductance regulator (
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Scientists around the world agree that global innovation is needed to address the shortage of effective antibiotics. Our Infection Research Initiative supports much-needed research and development, but new policies are needed to promote a sustainable, robust antibiotics pipeline and a marketplace that rewards innovation.
Through efficient study design, optimized clinical trial execution, and high-quality data, the Therapeutics Development Network (TDN) helps speed the delivery of new and better therapies to people with cystic fibrosis.
Some genetic diseases, such as cystic fibrosis, are caused by mutations in a single gene. A gene contains DNA “letters” that spell out the instructions to make a specific protein. When the protein isn't made correctly, it can lead to a cascade of problems.
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF.
The Drug Development Pipeline features an extensive list of drugs that are in development or approved for treating cystic fibrosis. For a drug or program to be shown on the pipeline, it must meet certain conditions.