Cystic fibrosis precision therapeutics: Emerging considerations

Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

(2019) Gitte Berkers et al.   Cell Reports

Correlating CFTR Function with Clinical Features to Inform Precision Treatment of Cystic Fibrosis

(2019) Allison F McCague et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Intestinal organoids to model Cystic Fibrosis

(2019) Peter van Mourik et al.   EUROPEAN RESPIRATORY JOURNAL

Considerations for Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease

(2018) Robert N. Schuck et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

CFTR modulator theratyping: Current status, gaps and future directions

(2018) John Paul Clancy et al.   Journal of Cystic Fibrosis

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

(2018) Dominic Keating et al.   NEW ENGLAND JOURNAL OF MEDICINE

VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

(2018) Jane C. Davies et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

(2017) Steven M. Rowe et al.   NEW ENGLAND JOURNAL OF MEDICINE

In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N-of-1 studies

(2017) Meghan E. McGarry et al.   PEDIATRIC PULMONOLOGY

Optimal correction of distinct CFTR folding mutants in rectal cystic fibrosis organoids

(2016) Johanna F. Dekkers et al.   EUROPEAN RESPIRATORY JOURNAL

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

(2016) Gudio Veit et al.   MOLECULAR BIOLOGY OF THE CELL

Classification of CFTR mutation classes

(2016) Frauke Stanke et al.   Lancet Respiratory Medicine

Progress in therapies for cystic fibrosis

(2016) Kris De Boeck et al.   Lancet Respiratory Medicine

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

(2015) Todd Gammie et al.   PLoS One

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1

(2013) Hong Yu Ren et al.   MOLECULAR BIOLOGY OF THE CELL

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

(2011) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation

(2010) Frank J. Accurso et al.   NEW ENGLAND JOURNAL OF MEDICINE

Alternative Splicing at a NAGNAG Acceptor Site as a Novel Phenotype Modifier

(2010) Alexandre Hinzpeter et al.   PLoS Genetics