Vertex Pharmaceuticals Inc. announced today that it submitted a New Drug Application to the U.S. Food and Drug Administration for the approval of the first triple-combination therapy for cystic fibrosis.
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Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
Phase 2 clinical trial results were announced today for a potential next-generation modulator to treat people with two copies of the most common cystic fibrosis mutation, F508del.
A new drug intended to make it easier to clear mucus from the lungs of people with cystic fibrosis showed promising results in a Phase 2 clinical trial. The drug, called SPX-101, blocks the overactive sodium channels that line CF lung cells.
Today Vertex Pharmaceuticals Inc. announced the two next-generation CFTR modulator candidates that will progress into Phase 3 clinical trials.
Yesterday, President Donald J. Trump signed the Right to Try bill into law. The House approved the bill last week and the Senate passed it last summer.
When I decided to study abroad in 1974, I also decided that I would not allow CF to stop me from living my life to the fullest.
When my 8-year-old daughter with CF found out that the disease is potentially fatal, I was grateful for her optimistic views on life and death. But deep down, I was saddened with the thought of a cure being so far away that I wouldn't be around to see it. Today, my fears have dwindled.