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.
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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.
6 min read
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.
3 min read