New funding awards include up to $2.6M to Eloxx Pharmaceuticals to identify potential therapies for CF nonsense mutations
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The Cystic Fibrosis Foundation is providing up to $5 million to develop a method to deliver a healthy copy of the CFTR gene into the lung cells of people with CF that is unlikely to trigger an immune system response.
Today we received the exciting news that the U.S. Food and Drug Administration has approved Orkambi for people with cystic fibrosis ages 12 and older who have two copies of the F508del gene mutation, the most common CF mutation.
The U.S. Food and Drug Administration announced today it has approved Kalydeco™ to treat people ages 6 and older who have one of eight additional cystic fibrosis mutations.
This morning, Vertex Pharmaceuticals announced the results from the Phase 3 clinical trials of ivacaftor (Kalydeco™) and lumacaftor (VX-809) in people with two copies of the F508del mutation.
The Cystic Fibrosis Foundation recently submitted comments to the National Institutes of Health on improving racial equity, diversity, and inclusion in the biomedical research workforce.