Cystic fibrosis: a review
T Ong, BW Ramsey - Jama, 2023 - jamanetwork.com
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 …
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in …
Increasing life expectancy in cystic fibrosis: Advances and challenges
KA McBennett, PB Davis, MW Konstan - Pediatric pulmonology, 2022 - Wiley Online Library
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 …
both quality of life and longevity for people living with this disease. In this article we describe …
A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele
ET Zemanick, JL Taylor-Cousar, J Davies… - American journal of …, 2021 - atsjournals.org
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 …
safe in patients≥ 12 years of age with cystic fibrosis and at least one F508del-CFTR (cystic …
Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function …
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 …
was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis …
The future of cystic fibrosis treatment: from disease mechanisms to novel therapeutic approaches
SY Graeber, MA Mall - The Lancet, 2023 - thelancet.com
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 …
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …
Cystic fibrosis modulator therapies
S Jia, JL Taylor-Cousar - Annual review of medicine, 2023 - annualreviews.org
Cystic fibrosis (CF) is an inherited multisystemic disease that can cause progressive
bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction …
bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction …
CFTR modulator therapy: transforming the landscape of clinical care in cystic fibrosis
JL Taylor-Cousar, PD Robinson, M Shteinberg… - The Lancet, 2023 - thelancet.com
Following discovery of the cystic fibrosis transmembrane conductance regulator (CFTR)
gene in 1989 and subsequent elucidation of the varied CFTR protein abnormalities that …
gene in 1989 and subsequent elucidation of the varied CFTR protein abnormalities that …
Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double‐blind, placebo‐controlled phase 1/2 clinical study
SM Rowe, JB Zuckerman, D Dorgan, J Lascano… - Journal of Cystic …, 2023 - Elsevier
Background MRT5005, a codon-optimized CFTR mRNA, delivered by aerosol in lipid
nanoparticles, was designed as a genotype-agnostic treatment for CF lung disease …
nanoparticles, was designed as a genotype-agnostic treatment for CF lung disease …
Real-world disparities and ethical considerations with access to CFTR modulator drugs: Mind the gap!
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 …
being for all at all ages, has particular relevance and implementation challenges amongst …
Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective
N Mayer-Hamblett, JP Clancy, R Jain… - The Lancet …, 2023 - thelancet.com
The growing use of modulator therapies aimed at restoring cystic fibrosis transmembrane
conductance regulator (CFTR) protein function in people with cystic fibrosis has …
conductance regulator (CFTR) protein function in people with cystic fibrosis has …