The first CF rat model, CFTR tm1sage (termed CFTR-/- or KO), was designed in collaboration with SAGE (now Horizon Discovery) and subsequently characterized at UAB and distributed to the CF community.
The CFTR Protein Distribution Core offers high-quality, full-length recombinant cystic fibrosis transmembrane conductance regulator (CFTR) proteins expressed in and purified from mammalian cells. Wild ...
The CFTR protein reaches the cell membrane but the ... with nasal potential difference within normal range cycle 1: 13 (57%; p = 0.0003) cycle 2: 9 (43%; p = 0.02) Weight change from baseline ...
Gene therapy consists of delivering a healthy copy of the CFTR gene to the lung cells of CF patients so that their bodies can ...
The US Food and Drug Administration has approved Alyftrek, a once-daily oral combination of vanzacaftor, tezacaftor, and ...
(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) Friday announced that the U.S. Food and Drug Administration (FDA) has approved Alyftrek for treatment of cystic fibrosis.
Vertex Pharmaceuticals (VRTX) announced that the U.S. Food and Drug Administration has approved Alyftrek, a once-daily next-in-class triple ...
“Our north star for more than 20 years has been to address the underlying cause of cystic fibrosis, treat more people with this disease, and bring more people to normal levels of CFTR function ...
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has ...
US drugmaker Vertex Pharmaceuticals’ shares were unmoved on Friday, despite receiving two new regulatory authorizations.