Sustained Benefit from Ivacaftor Demonstrated by Combining Clinical Trial and Cystic Fibrosis Patient Registry Data

Children and young adults with CF in the USA have better lung function compared with the UK

(2014) C. H. Goss et al.   THORAX

Accelerating implementation of biomedical research advances: critical elements of a successful 10 year Cystic Fibrosis Foundation healthcare delivery improvement initiative

(2014) Bruce C Marshall et al.   BMJ Quality & Safety

Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp- CFTR mutation: a phase 3, open-label extension study (PERSIST)

(2014) Edward F McKone et al.   Lancet Respiratory Medicine

Modifying disease in cystic fibrosis

(2013) Thida Ong et al.   CURRENT OPINION IN PULMONARY MEDICINE

Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D- CFTR mutation and preserved spirometry: a randomised controlled trial

(2013) Jane Davies et al.   Lancet Respiratory Medicine

Multi-ethnic reference values for spirometry for the 3–95-yr age range: the global lung function 2012 equations

(2012) Philip H. Quanjer et al.   EUROPEAN RESPIRATORY JOURNAL

Risk factors for rate of decline in FEV1 in adults with cystic fibrosis

(2012) Michael W. Konstan et al.   Journal of Cystic Fibrosis

Growth failure in children with cystic fibrosis

(2012) Alessandra Scaparrotta et al.   JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

(2011) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF MEDICINE

Clinical use of dornase alfa is associated with a slower rate of FEV1 decline in cystic fibrosis

(2011) Michael W. Konstan et al.   PEDIATRIC PULMONOLOGY

Design and powering of cystic fibrosis clinical trials using rate of FEV1 decline as an efficacy endpoint

(2010) M.W. Konstan et al.   Journal of Cystic Fibrosis

Propensity Score Methods

(2009) Donald B. Rubin   AMERICAN JOURNAL OF OPHTHALMOLOGY

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

(2009) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A critical appraisal of propensity-score matching in the medical literature between 1996 and 2003

(2007) Peter C. Austin   STATISTICS IN MEDICINE