Last week I travelled to D.C. to serve on a panel discussing the recent developments in precision medicine. I've got to say, it was pretty neat.
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Traveling with a kid with cystic fibrosis is better if you plan ahead, get organized, and create a schedule -- but always be able to roll with the punches if that schedule changes.
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.
I decided that I wanted to live on the Caribbean island that I grew up on. Although it takes some planning and work, I'm thriving there.
The Cystic Fibrosis Foundation awarded Talee Bio Inc. up to $4.5 million to develop methods for delivering potential gene therapy treatments to the lungs.
The U.S. Food and Drug Administration has approved the use of the triple-combination modulator elexacaftor/tezacaftor/ivacaftor (Trikafta™) for people with cystic fibrosis ages 12 and older who have at least one copy of the F508del mutation.
The Cystic Fibrosis Foundation awarded up to $5.1 million to TB Alliance to advance a drug discovery program in partnership with Johns Hopkins University. The effort will identify compounds with the potential to treat nontuberculous mycobacteria infections that are increasingly found among people with CF.
The U.S. Food and Drug Administration has approved the use of tezacaftor/ivacaftor (Symdeko®) for children with cystic fibrosis ages 6 to 11 with specific mutations.
Vertex Pharmaceuticals Inc. today announced that it will pursue FDA approval for the triple-combination therapy VX-445 (elexacaftor) plus tezacaftor/ivacaftor (Symdeko®).
Today, Vertex released positive preliminary clinical trial results for the potential triple-combination therapy VX-445 plus tezacaftor/ivacaftor (Symdeko®).