Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR
نویسندگان
چکیده
منابع مشابه
Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR.
RATIONALE Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with ivacaftor. OBJECTIVES To evaluate the safety and efficacy of tezacaftor monotherapy and of tezacaftor/ivacaftor combination therapy in subjects w...
متن کاملCystic Fibrosis and Cftr Gene
The early description of cystic fibrosis (CF) dates back to late 30s. In 1936, Fanconi identified the association between the “congenital CF of the pancreas and broncheactasis" shortly followed by Andersen who in 1938 gave the complete anatomopathologic description of CF. In 1953, Di Sant'Agnese described an excess of sodium chloride in the sweat of children affected by CF. This discovery short...
متن کاملCystic Fibrosis and CFTR Gene
I. Background II. Incidence III. Clinical Manifestations IV. Diagnosis V. CFTR gene and its mutations V.
متن کاملCFTR: cystic fibrosis and beyond.
Cystic fibrosis (CF) remains the most common fatal hereditary lung disease. The discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene 25 years ago set the stage for: 1) unravelling the molecular and cellular basis of CF lung disease; 2) the generation of animal models to study in vivo pathogenesis; and 3) the development of mutation-specific therapies that are now bec...
متن کاملCystic Fibrosis and Cftr Gene
The early description of cystic fibrosis (CF) dates back to late 30s. In 1936, Fanconi identified the association between the “congenital CF of the pancreas and broncheactasis" shortly followed by Andersen who in 1938 gave the complete anatomopathologic description of CF. In 1953, Di Sant'Agnese described an excess of sodium chloride in the sweat of children affected by CF. This discovery short...
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ژورنال
عنوان ژورنال: American Journal of Respiratory and Critical Care Medicine
سال: 2018
ISSN: 1073-449X,1535-4970
DOI: 10.1164/rccm.201704-0717oc