Importance Cystic fibrosis, a genetic disorder defined by variants in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in …

Since the first description of cystic fibrosis in 1938, there have been significant advances in
both quality of life and longevity for people living with this disease. In this article we describe …

Rationale: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and
safe in patients≥ 12 years of age with cystic fibrosis and at least one F508del-CFTR (cystic …

Rationale: The triple-combination regimen elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA)
was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis …

With the 2019 breakthrough in the development of highly effective modulator therapy
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …

Cystic fibrosis (CF) is an inherited multisystemic disease that can cause progressive
bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction …

Following discovery of the cystic fibrosis transmembrane conductance regulator (CFTR)
gene in 1989 and subsequent elucidation of the varied CFTR protein abnormalities that …

Background MRT5005, a codon-optimized CFTR mRNA, delivered by aerosol in lipid
nanoparticles, was designed as a genotype-agnostic treatment for CF lung disease …

The third Sustainable Development Goal (SDG), to ensure healthy lives and promote well-
being for all at all ages, has particular relevance and implementation challenges amongst …

The growing use of modulator therapies aimed at restoring cystic fibrosis transmembrane
conductance regulator (CFTR) protein function in people with cystic fibrosis has …