Ontology highlight
ABSTRACT:
SUBMITTER: Liessi N
PROVIDER: S-EPMC7455125 | biostudies-literature | 2020 Aug
REPOSITORIES: biostudies-literature
Liessi Nara N Pesce Emanuela E Braccia Clarissa C Bertozzi Sine Mandrup SM Giraudo Alessandro A Bandiera Tiziano T Pedemonte Nicoletta N Armirotti Andrea A
JCI insight 20200820 16
In recent years, a number of drugs have been approved for the treatment of cystic fibrosis (CF). Among them, newly released Trikafta, a combination of 3 drugs (VX-661/VX-445/VX-770), holds great promise to radically improve the quality of life for a large portion of patients with CF carrying 1 copy of F508del, the most frequent CF transmembrane conductance regulator (CFTR) mutation. Currently available disease-modifying CF drugs work by rescuing the function of the mutated CFTR anion channel. Re ...[more]