The Impact of Highly Effective CFTR Modulators on Growth and Nutrition Status

Genetic potential and height velocity during childhood and adolescence do not fully account for shorter stature in cystic fibrosis

(2020) Zofia N. Zysman-Colman et al.   PEDIATRIC RESEARCH

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

Nutritional Status in Childhood as a Prognostic Factor in Patients with Cystic Fibrosis

(2019) Moshe Ashkenazi et al.   LUNG

An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2–5 years (KLIMB)

(2019) Margaret Rosenfeld et al.   Journal of Cystic Fibrosis

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

(2019) Harry G M Heijerman et al.   LANCET

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

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

Growth and Nutrition in Cystic Fibrosis

(2019) Jefferson N. Brownell et al.   SEMINARS IN RESPIRATORY AND CRITICAL CARE MEDICINE

Energy Balance and Mechanisms of Weight Gain with Ivacaftor Treatment of Cystic Fibrosis Gating Mutations

(2018) Virginia A. Stallings et al.   JOURNAL OF PEDIATRICS

Association between body composition and pulmonary function in children and young people with cystic fibrosis

(2018) Patrizia Calella et al.   NUTRITION

Ivacaftor treatment of cystic fibrosis in children aged 12 to

(2018) Margaret Rosenfeld et al.   Lancet Respiratory Medicine

IVACAFTOR restores FGF19 regulated bile acid homeostasis in cystic fibrosis patients with an S1251N or a G551D gating mutation

(2018) Ivo P. van de Peppel et al.   Journal of Cystic Fibrosis

Bile acid homeostasis in gastrointestinal and metabolic complications of cystic fibrosis

(2018) Ivo P. van de Peppel et al.   Journal of Cystic Fibrosis

Impact of CFTR modulation with Ivacaftor on Gut Microbiota and Intestinal Inflammation

(2018) Chee Y. Ooi et al.   Scientific Reports

Pancreatic insufficiency in Cystic Fibrosis

(2017) Vikesh K. Singh et al.   Journal of Cystic Fibrosis

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

Poster Session Abstracts

(2017)   PEDIATRIC PULMONOLOGY

Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor

(2017) Michael S. Stalvey et al.   PEDIATRICS

Effects of Diagnosis by Newborn Screening for Cystic Fibrosis on Weight and Length in the First Year of Life

(2017) Daniel H. Leung et al.   JAMA Pediatrics

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR : a randomised, placebo-controlled phase 3 trial

(2017) Felix Ratjen et al.   Lancet Respiratory Medicine

Impact of CFTR Modulation on Intestinal pH, Motility and Clinical Outcomes in Patients With Cystic Fibrosis and the G551D Mutation

(2017) Daniel Gelfond et al.   Clinical and Translational Gastroenterology

ESPEN-ESPGHAN-ECFS guidelines on nutrition care for infants, children, and adults with cystic fibrosis

(2016) Dominique Turck et al.   CLINICAL NUTRITION

Disrupted progression of the intestinal microbiota with age in children with cystic fibrosis

(2016) Shaun Nielsen et al.   Scientific Reports

Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2–5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study

(2016) Jane C Davies et al.   Lancet Respiratory Medicine

Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor

(2015) Drucy Borowitz et al.   DIGESTIVE DISEASES AND SCIENCES

Improvement of Hepatic Steatosis in Cystic Fibrosis With Ivacaftor Therapy

(2015) Don Hayes et al.   JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION

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

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

Intestinal Lesions Are Associated with Altered Intestinal Microbiome and Are More Frequent in Children and Young Adults with Cystic Fibrosis and Cirrhosis

(2015) Thomas Flass et al.   PLoS One

Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

(2014) Steven M. Rowe et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Pancreatic enzyme replacement therapy for pancreatic exocrine insufficiency in the 21stcentury

(2014) Tony Trang   WORLD JOURNAL OF GASTROENTEROLOGY

Body Composition and Pulmonary Function in Cystic Fibrosis

(2014) Saba Sheikh et al.   Frontiers in Pediatrics

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with aG551DMutation

(2013) Jane C. Davies et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Osteoblast CFTR Inactivation Reduces Differentiation and Osteoprotegerin Expression in a Mouse Model of Cystic Fibrosis-Related Bone Disease

(2013) Michael S. Stalvey et al.   PLoS One

Update of Faecal Markers of Inflammation in Children with Cystic Fibrosis

(2012) Jung M. Lee et al.   MEDIATORS OF INFLAMMATION

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

Longitudinal Analysis of Resting Energy Expenditure in Patients with Cystic Fibrosis

(2007) Annabel Magoffin et al.   JOURNAL OF PEDIATRICS