Cystic Fibrosis

Improved glucose tolerance after initiation of Elexacaftor / Tezacaftor / Ivacaftor in adults with cystic fibrosis

(2023) Carolin Steinack et al.   Journal of Cystic Fibrosis

Longitudinal effects of elexacaftor/tezacaftor/ivacaftor on liver tests at a large single adult cystic fibrosis centre

(2023) Daniel H Tewkesbury et al.   Journal of Cystic Fibrosis

Effects of elexacaftor/tezacaftor/ivacaftor therapy in children with cystic fibrosis – a comprehensive assessment using lung clearance index, spirometry, and functional and structural lung MRI

(2023) Carmen Streibel et al.   Journal of Cystic Fibrosis

Role of inhaled antibiotics in the era of highly effective CFTR modulators

(2023) J. Stuart Elborn et al.   European Respiratory Review

Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2 Through 5 Years with Cystic Fibrosis and at Least One F508del Allele

(2023) Jennifer L. Goralski et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

The Impact of Highly Effective Cystic Fibrosis Transmembrane Conductance Regulator Modulators on the Health of Female Subjects With Cystic Fibrosis

(2023) Jennifer L. Taylor-Cousar et al.   CLINICAL THERAPEUTICS

Cost-effectiveness analysis of genetic tools to predict treatment response in patients with cystic fibrosis

(2023) Yeva Sahakyan et al.   Journal of Cystic Fibrosis

Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double‐blind, placebo‐controlled phase 1/2 clinical study

(2023) S.M. Rowe et al.   Journal of Cystic Fibrosis

Effects of elexacaftor/tezacaftor/ivacaftor therapy on mental health of patients with cystic fibrosis

(2023) Linus Piehler et al.   Frontiers in Pharmacology

Assays of CFTR Function In Vitro, Ex Vivo and In Vivo

(2022) Anabela S. Ramalho et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Sustained effectiveness of elexacaftor-tezacaftor-ivacaftor in lung transplant candidates with cystic fibrosis

(2022) Clémence Martin et al.   Journal of Cystic Fibrosis

Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on Lung Clearance Index and Magnetic Resonance Imaging in Patients with Cystic Fibrosis and One or Two F508del Alleles

(2022) Simon Y. Graeber et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Obesity in Cystic fibrosis: prevalence, trends and associated factors data from the US cystic fibrosis foundation patient registry

(2022) Sylvia Szentpetery et al.   Journal of Cystic Fibrosis

A case report of CFTR modulator administration via carrier mother to treat meconium ileus in a F508del homozygous fetus

(2022) Sylvia Szentpetery et al.   Journal of Cystic Fibrosis

Caution advised in the use of CFTR modulator treatment for individuals harboring specific CFTR variants

(2022) Karen S. Raraigh et al.   Journal of Cystic Fibrosis

Use of elexacaftor/tezacaftor/ivacaftor among cystic fibrosis lung transplant recipients

(2022) Kathleen J. Ramos et al.   Journal of Cystic Fibrosis

The effect of inhaled hypertonic saline on lung structure in children aged 3–6 years with cystic fibrosis (SHIP-CT): a multicentre, randomised, double-blind, controlled trial

(2022) Harm A W M Tiddens et al.   Lancet Respiratory Medicine

Association of Body Mass Index With Clinical Outcomes in Patients With Cystic Fibrosis

(2022) Rita Nagy et al.   JAMA Network Open

Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3B, Randomized, Placebo-Controlled Study

(2022) Marcus A Mall et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Current state of CFTR modulators for treatment of Cystic Fibrosis

(2022) Katherine A. Despotes et al.   CURRENT OPINION IN PHARMACOLOGY

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

(2022) Denise E. Sabatino et al.   MOLECULAR THERAPY

One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies

(2022) Marjolein M. Ensinck et al.   Cells

Biologic drugs in treating allergic bronchopulmonary aspergillosis in patients with cystic fibrosis: a systematic review

(2022) Sara Manti et al.   European Respiratory Review

Progress in Respiratory Gene Therapy

(2022) Gerry McLachlan et al.   HUMAN GENE THERAPY

Beyond borders: cystic fibrosis survival between Australia, Canada, France and New Zealand

(2022) Adèle Coriati et al.   THORAX

Treatment Preference Among People With Cystic Fibrosis

(2022) Rory A. Cameron et al.   CHEST

The Changing Epidemiology of Cystic Fibrosis

(2022) Pierre-Régis Burgel et al.   CHEST

Update in Advancing the Gastrointestinal Frontier in Cystic Fibrosis

(2022) Christopher Vélez et al.   CLINICS IN CHEST MEDICINE

Contemporary cystic fibrosis incidence rates in Canada and the United States

(2022) Anne L. Stephenson et al.   Journal of Cystic Fibrosis

Variation in cystic fibrosis newborn screening algorithms in the United States

(2022) Maryann R. Rehani et al.   PEDIATRIC PULMONOLOGY

Beyond the Lungs—Emerging Challenges in Adult Cystic Fibrosis Care

(2022) Siobhain Mulrennan et al.   SEMINARS IN RESPIRATORY AND CRITICAL CARE MEDICINE

Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials

(2022) Nicole Mayer-Hamblett et al.   Lancet Respiratory Medicine

Cystic Fibrosis Patients of Minority Race and Ethnicity Less Likely Eligible for CFTR Modulators Based on CFTR Genotype

(2021) Meghan E. McGarry et al.   PEDIATRIC PULMONOLOGY

Determinants of lung disease progression measured by lung clearance index in children with cystic fibrosis

(2021) Sanja Stanojevic et al.   EUROPEAN RESPIRATORY JOURNAL

Survival and lung transplant outcomes for individuals with advanced cystic fibrosis lung disease in the United States and Canada: an analysis of national registries

(2021) Kathleen J. Ramos et al.   CHEST

Anti-Inflammatory Influences of Cystic Fibrosis Transmembrane Conductance Regulator Drugs on Lung Inflammation in Cystic Fibrosis

(2021) Kiera H. Harwood et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation

(2021) Yifat S. Oren et al.   Journal of Cystic Fibrosis

Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes

(2021) Peter J. Barry et al.   NEW ENGLAND JOURNAL OF MEDICINE

Magnetic resonance imaging of cystic fibrosis: Multi-organ imaging in the age of CFTR modulator therapies

(2021) Kimberly McBennett et al.   Journal of Cystic Fibrosis

Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial

(2021) Sivagurunathan Sutharsan et al.   Lancet Respiratory Medicine

Lung Transplantation for Cystic Fibrosis

(2020) Jonathan C Yeung et al.   JOURNAL OF HEART AND LUNG TRANSPLANTATION

Cystic fibrosis-related diabetes: an update on pathophysiology, diagnosis, and treatment

(2020) Crésio Alves et al.   JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM

Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

(2020) Renée V. E. Dagenais et al.   Journal of Clinical Medicine

In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis

(2019) Xingshen Sun et al.   Science Translational Medicine

Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries

(2019) Nataliya Volkova et al.   Journal of Cystic Fibrosis

Inhaled hypertonic saline in preschool children with cystic fibrosis (SHIP): a multicentre, randomised, double-blind, placebo-controlled trial

(2019) Felix Ratjen et al.   Lancet Respiratory Medicine

The future of cystic fibrosis care: a global perspective

(2019) Scott C Bell et al.   Lancet Respiratory Medicine

Long-term effect of CFTR modulator therapy on airway nitric oxide

(2019) Hartmut Grasemann et al.   EUROPEAN RESPIRATORY JOURNAL

Cystic fibrosis bone disease treatment: Current knowledge and future directions

(2019) Melissa S. Putman et al.   Journal of Cystic Fibrosis

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

(2019) Peter G. Middleton et al.   NEW ENGLAND JOURNAL OF MEDICINE

Changes in Lung Clearance Index in Preschool Cystic Fibrosis Patients Treated with Ivacaftor

(2018) Felix Ratjen et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Interleukin-1 is associated with inflammation and structural lung disease in young children with cystic fibrosis

(2018) Samuel T. Montgomery 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

Genomic sequencing in cystic fibrosis newborn screening: what works best, two-tier predefined CFTR mutation panels or second-tier CFTR panel followed by third-tier sequencing?

(2017) Robert J Currier et al.   GENETICS IN MEDICINE

Diagnosis of Cystic Fibrosis: Consensus Guidelines from the Cystic Fibrosis Foundation

(2017) Philip M. Farrell et al.   JOURNAL OF PEDIATRICS

CFTR Modulator Therapy for Cystic Fibrosis

(2017) Hartmut Grasemann   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

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

(2017) Jennifer L. Taylor-Cousar et al.   NEW ENGLAND JOURNAL OF MEDICINE

Electrolyte transport properties in distal small airways from cystic fibrosis pigs with implications for host defense

(2016) Xiaopeng Li et al.   AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

Role of CFTR in epithelial physiology

(2016) Vinciane Saint-Criq et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Body composition and lung function in cystic fibrosis and their association with adiposity and normal-weight obesity

(2016) Jessica A. Alvarez et al.   NUTRITION

Cystic Fibrosis Screen Positive, Inconclusive Diagnosis (CFSPID): A new designation and management recommendations for infants with an inconclusive diagnosis following newborn screening

(2015) A. Munck et al.   Journal of Cystic Fibrosis

Origins of Cystic Fibrosis Lung Disease

(2015) David A. Stoltz et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

International Committee on Mental Health in Cystic Fibrosis: Cystic Fibrosis Foundation and European Cystic Fibrosis Society consensus statements for screening and treating depression and anxiety

(2015) Alexandra L Quittner et al.   THORAX

Cystic fibrosis

(2015) Felix Ratjen et al.   Nature Reviews Disease Primers

Aquagenic Wrinkling of the Palms in a Patient with Cystic Fibrosis

(2013) Hartmut Grasemann et al.   NEW ENGLAND JOURNAL OF MEDICINE

Early lung disease in cystic fibrosis

(2013) Hartmut Grasemann et al.   Lancet Respiratory Medicine

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

(2012) Alejandro A. Pezzulo et al.   NATURE

Recommendations for the classification of diseases as CFTR-related disorders

(2011) C. Bombieri et al.   Journal of Cystic Fibrosis

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

Type of CFTR Mutation Determines Risk of Pancreatitis in Patients With Cystic Fibrosis

(2010) Chee Y. Ooi et al.   GASTROENTEROLOGY

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

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

Cystic Fibrosis Pulmonary Guidelines

(2009) Patrick A. Flume et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

The Effect of Chronic Infection With Aspergillus fumigatus on Lung Function and Hospitalization in Patients With Cystic Fibrosis

(2009) Reshma Amin et al.   CHEST

Pulmonary Complications of Cystic Fibrosis

(2009) Patrick A Flume   Respiratory Care