A fixed-dose combination tablet of the cystic fibrosis transmembrane conductance regulator
(CFTR) corrector tezacaftor and the CFTR potentiator ivacaftor with the next-generation …

Two mycobacteriophages were administered intravenously to a male with treatment-
refractory Mycobacterium abscessus pulmonary infection and severe cystic fibrosis lung …

Several placebo-controlled trials have been recently published evaluating novel therapies
targeting the defective CFTR protein. This systematic review examines the clinical efficacy …

Background The next-generation cystic fibrosis transmembrane conductance regulator
(CFTR) corrector VX-659, in triple combination with tezacaftor and ivacaftor (VX-659 …

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator
gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel …

Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the
Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane …

Genetic and rare diseases (GARDs) affect more than 350 million patients worldwide and
remain a significant challenge in the clinic. Hence, continuous efforts have been made to …

Childhood asthma remains a multifactorial disease with heterogeneous clinical phenotype
and complex genetic inheritance. The primary aim of asthma management is to achieve …

Introduction Elexacaftor/tezacaftor/ivacaftor (ETI) treatment is associated with significant
improvement in lung function in people with cystic fibrosis (pwCF); however, some patients …

The new CFTR modulator combination, elexacaftor/tezacaftor/ivacaftor (Trikafta) was
approved by the FDA in October 2019 for treatment of Cystic Fibrosis in patients 6 years of …