Vertex Pharmaceuticals Incorporated Common Stock (VRTX) Quote & Summary Data
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From Wikipedia, the free encyclopedia. Jeffrey Leiden , M. Retrieved 24 June Retrieved 25 June One Company's Quest for the Perfect Drug. Food and Drug Administration. A Price Too High to Pay? Charity and Industry Partner for Profit". Retrieved July 19, Milla; Mark Pian; et al. Business data for Vertex Pharmaceuticals: Finance Reuters SEC filings.
Pharmaceutical companies of the United States. A Phase 3 clinical study is underway in children up to 24 months of age, who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator CFTR gene: Ivacaftor is designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface.
The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage. A Phase 3 clinical study was completed in children ages 2 through 5 with cystic fibrosis who have two copies of the Fdel mutation in the CFTR gene. A Phase 3, rollover long-term safety study is underway in children aged 2 years and older with cystic fibrosis who have two copies of the Fdel mutation in the CFTR gene.
Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the Fdel CFTR protein, and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.
This helps improve the flow of salt and water in to and out of cells. To learn more, visit clinicaltrials. We have completed other Phase 3 clinical studies in people with these mutations ages 12 and older. Tezacaftor is designed to increase the amount of mature protein at the cell surface by aiding the processing and trafficking of the CFTR protein, and ivacaftor is designed to enhance the channel open probability or gating of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.
VX and tezacaftor VX are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the Fdel CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.
Two Phase 3 studies of VX, tezacaftor and ivacaftor as an investigational triple combination regimen are underway in people with CF ages 12 and older. To learn more, visit ClinicalTrials. A second Phase 3 study is underway in patients who have two copies of the Fdel mutation. For a list of the minimal function mutations currently included in the study, click here. Cystic fibrosis CF is a rare, life-threatening genetic disease affecting approximately 75, people in North America, Europe and Australia.
There is also a Phase 2 clinical investigational study of VX in people ages 12 and older who have primary ciliary dyskinesia PCD. There is also a Phase 2 clinical investigational study of VX in people ages 12 and older who have primary ciliary dyskinesia PCD , a rare genetic disease that results in a loss of function in key ciliary proteins. The defective proteins lead to dysfunctional beating of cilia on the surface of cells, especially in the lungs, where the accumulation of mucus can lead to chronic lung infections, bronchiectasis and progressive lung function decline.
Intraday Last 52 Weeks High: Data reflects weightings calculated at the beginning of each month. Data is subject to change. Company Description as filed with the SEC We invest in scientific innovation to create transformative medicines for serious diseases.
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