Emerging drugs for cystic fibrosis

The Impact of MRSA on Lung Function in Patients with Cystic Fibrosis

(2013) Gregory S. Sawicki et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Lung Clearance Index as an Outcome Measure for Clinical Trials in Young Children with Cystic Fibrosis. A Pilot Study Using Inhaled Hypertonic Saline

(2013) Padmaja Subbarao et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function

(2013) Fredrick Van Goor et al.   Journal of Cystic Fibrosis

Adenoviral Gene Transfer Corrects the Ion Transport Defect in the Sinus Epithelia of a Porcine CF Model

(2013) Andrea E Potash et al.   MOLECULAR THERAPY

Mechanism-based corrector combination restores ΔF508-CFTR folding and function

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis: Table 1

(2013) Eric W F W Alton et al.   THORAX

Phase II studies of nebulised Arikace in CF patients withPseudomonas aeruginosainfection

(2013) J P Clancy et al.   THORAX

Assessment of F/HN-Pseudotyped Lentivirus as a Clinically Relevant Vector for Lung Gene Therapy

(2012) Uta Griesenbach et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

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

(2012) Patrick A. Flume et al.   CHEST

Novel Therapeutic Uses of Alpha-1 Antitrypsin: A Window to the Future

(2012) Adam Wanner et al.   COPD-Journal of Chronic Obstructive Pulmonary Disease

Inhaled Hypertonic Saline in Infants and Children Younger Than 6 Years With Cystic Fibrosis

(2012) Margaret Rosenfeld et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Treatment of lung infection in patients with cystic fibrosis: Current and future strategies

(2012) Gerd Döring et al.   Journal of Cystic Fibrosis

Ivacaftor potentiation of multiple CFTR channels with gating mutations

(2012) Haihui Yu et al.   Journal of Cystic Fibrosis

Pooled analysis of two large randomised phase III inhaled mannitol studies in cystic fibrosis

(2012) Diana Bilton et al.   Journal of Cystic Fibrosis

Reduced airway surface pH impairs bacterial killing in the porcine cystic fibrosis lung

(2012) Alejandro A. Pezzulo et al.   NATURE

Safety, efficacy and convenience of colistimethate sodium dry powder for inhalation (Colobreathe DPI) in patients with cystic fibrosis: a randomised study

(2012) Antje Schuster et al.   THORAX

Levofloxacin Inhalation Solution (MP-376) in Patients with Cystic Fibrosis withPseudomonas aeruginosa

(2011) David E. Geller et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Long-Term Inhaled Dry Powder Mannitol in Cystic Fibrosis

(2011) Moira L. Aitken et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Fosfomycin/Tobramycin for Inhalation in Patients with Cystic Fibrosis withPseudomonasAirway Infection

(2011) Bruce C. Trapnell et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Pharmacokinetics and Safety of MP-376 (Levofloxacin Inhalation Solution) in Cystic Fibrosis Subjects

(2011) David E. Geller et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

(2011) M. Wilschanski et al.   EUROPEAN RESPIRATORY JOURNAL

Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study

(2011) D. Bilton et al.   EUROPEAN RESPIRATORY JOURNAL

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

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

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

Tobramycin Inhalation Powder™: a novel drug delivery system for treating chronicPseudomonas aeruginosainfection in cystic fibrosis

(2011) Michael D Parkins et al.   Expert Review of Respiratory Medicine

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

(2010) Isabelle Sermet-Gaudelus et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

To the Editor

(2010) Alan L. Coates   Journal of Aerosol Medicine and Pulmonary Drug Delivery

Inhaled mannitol in patients with cystic fibrosis: A randomised open-label dose response trial

(2010) Alejandro Teper et al.   Journal of Cystic Fibrosis

Safety, efficacy and convenience of tobramycin inhalation powder in cystic fibrosis patients: The EAGER trial

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

Symposium Summaries

(2010)   PEDIATRIC PULMONOLOGY

Tobramycin inhalation powder for P. aeruginosa infection in cystic fibrosis: The EVOLVE trial

(2010) Michael W. Konstan et al.   PEDIATRIC PULMONOLOGY

Respiratory Microbiology of Patients With Cystic Fibrosis in the United States, 1995 to 2005

(2009) Samiya Razvi et al.   CHEST

Lung deposition of inhaled  1-proteinase inhibitor in cystic fibrosis and  1-antitrypsin deficiency

(2009) P. Brand et al.   EUROPEAN RESPIRATORY JOURNAL

A Gamma Scintigraphy Study to Investigate Lung Deposition and Clearance of Inhaled Amikacin-Loaded Liposomes in Healthy Male Volunteers

(2009) Jeffry Weers et al.   Journal of Aerosol Medicine and Pulmonary Drug Delivery

Safety/Efficacy of Inhaled Human Alpha-1 Antitrypsin (AAT) in CF: A Phase II Clinical Study

(2009) E. Kerem et al.   Journal of Cystic Fibrosis

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

Aerosol Antibiotics in Cystic Fibrosis

(2009) David E Geller   Respiratory Care

Comparison of inhaled mannitol, daily rhDNase and a combination of both in children with cystic fibrosis: a randomised trial

(2009) C Minasian et al.   THORAX

Characterization of Nebulized Liposomal Amikacin (Arikace™) as a Function of Droplet Size

(2008) Zhili Li et al.   Journal of Aerosol Medicine and Pulmonary Drug Delivery

Biofilm penetration, triggered release and in vivo activity of inhaled liposomal amikacin in chronic Pseudomonas aeruginosa lung infections

(2008) P. Meers et al.   JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY

Single-dose pharmacokinetics of aerosol MP-376 (levofloxacin solution for inhalation) in cystic fibrosis patients: PK-PD implications

(2008) P.C. Griffith 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

The impact of incident methicillin resistant Staphylococcus aureus detection on pulmonary function in cystic fibrosis

(2008) Gregory S. Sawicki et al.   PEDIATRIC PULMONOLOGY

The science of aerosol delivery in cystic fibrosis

(2008) David E. Geller   PEDIATRIC PULMONOLOGY

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

TMEM16A, A Membrane Protein Associated with Calcium-Dependent Chloride Channel Activity

(2008) A. Caputo et al.   SCIENCE