Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis

The changing face of the exocrine pancreas in cystic fibrosis: pancreatic sufficiency, pancreatitis and genotype

(2009) Jarosław Walkowiak et al.   EUROPEAN JOURNAL OF GASTROENTEROLOGY & HEPATOLOGY

Cystic fibrosis

(2009) Brian P O'Sullivan et al.   LANCET

Impact of different chloride and glucose solutions on nasal potential difference

(2009) Hugh H. House et al.   PEDIATRIC PULMONOLOGY

CFTR Delivery to 25% of Surface Epithelial Cells Restores Normal Rates of Mucus Transport to Human Cystic Fibrosis Airway Epithelium

(2009) Liqun Zhang et al.   PLOS BIOLOGY

PTC124 induces CFTR full-length production and activity in children with nonsense-mutation-mediated CF

(2008) I. Sermet-Gaudelus et al.   Journal of Cystic Fibrosis

The prevalence of cystic fibrosis in the European Union

(2008) Philip M. Farrell   Journal of Cystic Fibrosis

High treatment burden in adults with cystic fibrosis: Challenges to disease self-management

(2008) Gregory S. Sawicki et al.   Journal of Cystic Fibrosis

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial

(2008) Eitan Kerem et al.   LANCET

Assessment of the CFTR and ENaC association

(2008) Bakhrom K. Berdiev et al.   Molecular BioSystems

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model

(2008) M. Du et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA