Cystic fibrosis in an era of genomically guided therapy

Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

(2012) Patrick A. Flume et al.   CHEST

Cystic fibrosis drug Vertex's latest triumph

(2012) Heidi Ledford   NATURE BIOTECHNOLOGY

The Prevalence and Cost of Unapproved Uses of Top-Selling Orphan Drugs

(2012) Aaron S. Kesselheim et al.   PLoS One

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

Pricing and reimbursement of orphan drugs: the need for more transparency

(2011) Steven Simoens   Orphanet Journal of Rare Diseases

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for theF508del-CFTRmutation

(2011) J P Clancy et al.   THORAX

Therapeutics Development for Cystic Fibrosis: A Successful Model for a Multisystem Genetic Disease

(2010) Melissa A. Ashlock et al.   Annual Review of Medicine

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

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

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

The Promise of a Cure: 20 Years and Counting

(2009) J. Couzin-Frankel   SCIENCE

Cystic fibrosis gene therapy: successes, failures and hopes for the future

(2009) Uta Griesenbach et al.   Expert Review of Respiratory Medicine

Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy

(2008) Uta Griesenbach et al.   ADVANCED DRUG DELIVERY REVIEWS

The prevalence of cystic fibrosis in the European Union

(2008) Philip M. Farrell   Journal of Cystic Fibrosis