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Clinical trials are critical to developing new treatments for CF. So, how exactly does an experimental drug become an approved therapy?
Choosing to volunteer in a
Cystic fibrosis is caused by mutations in both copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Scientists are examining whether it is possible to correct the mutations through a process called gene editing.
Clinical trials that test potential drugs and therapies in people with cystic fibrosis are a major part of CF research. They take place at Cystic Fibrosis Foundation-accredited care centers all over the United States and enroll people with CF of all ages.
When you are deciding whether to join a clinical trial, you should consider logistics.
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.
Robust funding for the National Institutes of Health helps ensure innovation in basic research and a full pipeline of cystic fibrosis therapies. A well-resourced U.S. Food and Drug Administration helps advance therapies that are safe and effective.
Stem cells are a special type of
Multiple Principal Investigator Clinical Awards are offered to provide support for multi-investigator-initiated clinical research projects that have the potential to make an important contribution to the Cystic Fibrosis Foundation’s mission.