Researchers at the Children’s Hospital Colorado Breathing Institute, the largest pediatric cystic fibrosis (CF) clinical care center in the U.S., participated in the pivotal international multi-center Phase 3 clinical trial, sponsored by Vertex Pharmaceuticals Incorporated, leading to FDA approval of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor), a highly effective CF treatment, for patients ages 6 through 11 who have at least one copy of the...
Tag: <span>CFTR</span>
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Discovery could improve cystic fibrosis treatment
Researchers exploring the effects of a long-standing treatment for cystic fibrosis have discovered a potential new target for drugs to treat the disease, which has no cure and typically cuts decades off the lives of patients. Cystic fibrosis affects the lungs and other organs, primarily by making the mucus that lines the lungs viscous, clogging...
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Fighting cystic fibrosis with CFTR amplifier
CAMBRIDGE, Mass.—Proteostasis Therapeutics Inc. (PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, announced a few days ago that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PTI-428, the Company’s cystic fibrosis transmembrane conductance...