Results 221 to 230 of about 1,459,532 (261)

Changes in fecal elastase-1 following initiation of CFTR modulator therapy in pediatric patients with cystic fibrosis.

Journal of Cystic Fibrosis, 2023
BACKGROUND Improvement in exocrine pancreatic function in persons with CF (pwCF) on cystic fibrosis transmembrane conductance regulator (CFTR) modulators has been documented in clinical trials using fecal pancreatic elastase-1 (FE-1).
K. Stephenson, Abby J. Lingle, Kelly A. Baumberger, E. Dellon, Charles R. Esther, Ellen Meier, C. Oermann, Vivek K. Shenoy, N. Smith, N. Wimmer, S. Duehlmeyer, Charissa W. Kam, Cameron J. McKinzie, M. O. Poisson, E. Elson   +14 more
semanticscholar   +1 more source

The impact of CFTR modulators on bone health

Pediatric Pulmonology, 2022
We are hopeful that CTFR modulation does have a positive impact of bone health, but we have concerns about this study design being used to support the hypothesis.
Elizabeth, Clarke, Fiona, Moore, Andrew, Gallagher, Stephen, Thomson   +3 more
openaire   +2 more sources

Improvement of lipid and lipoprotein profiles in children and adolescents with cystic fibrosis on CFTR modulator therapy.

Journal of Cystic Fibrosis, 2023
BACKGROUND Association of a high-fat diet with increased risks of cardiovascular disease (CVD) and type 2 diabetes, has prompted evaluation of lipids in people with CF (pwCF).
T. Yuzyuk, C. McDonald, L. Zuromski, I. De Biase, Lisa Johnson, N. Williams, S. Meihls, F. Asfour   +7 more
semanticscholar   +1 more source

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

Journal of Cystic Fibrosis, 2022
We report elexacaftor-tezacaftor-ivacaftor (ETI) treatment of a F508del carrier who was pregnant with a F508del homozygous fetus. At 23-weeks gestation meconium ileus (MI) was evident on ultrasound including dilated, hyperechoic bowel, which persisted on
S. Szentpetery, K. Foil, S. Hendrix, S. Gray, C. Mingora, Barbara Head, Donna Johnson, P. Flume   +7 more
semanticscholar   +1 more source

Real world outcomes of CFTR modulator therapy in Australian adults and children.

Pulmonary Pharmacology & Therapeutics, 2023
BACKGROUND Recent advances in CFTR modulator therapy have the potential to change the face of cystic fibrosis (CF). This retrospective observational study describes real world experience of the four available CFTR modulators in adults and children with ...
Stephanie L Kuek, A. McCullagh, E. Paul, D. Armstrong   +3 more
semanticscholar   +1 more source

Effect of Triple Combination CFTR Modulator Therapy on Sleep in Adult Patients with Cystic Fibrosis

Respiration, 2022
Background: Sleep-disordered breathing (SDB) and disturbed sleep are common, often underrecognized, comorbidities in people with cystic fibrosis (pwCF). Objectives: We studied the effect of CFTR triple combination therapy elexacaftor/tezacaftor/ivacaftor
M. Welsner, T. Schulte, S. Dietz-Terjung, G. Weinreich, F. Stehling, C. Taube, S. Strassburg, C. Schoebel, S. Sutharsan   +8 more
semanticscholar   +1 more source

WNK1 and WNK4 modulate CFTR activity

Biochemical and Biophysical Research Communications, 2007
The cystic fibrosis transmembrane conductance regulator (CFTR) is an ATP-gated chloride channel. WNK kinases are widely expressed modulators of ion transport. WNK1 and WNK4, two WNK kinases that are mutated in familial hyperkalemic hypertension (FHHt), are co-expressed with CFTR in several organs, raising the possibility that WNK kinases might alter ...
Chao-Ling, Yang, Xuehong, Liu, Alex, Paliege, Xiaoman, Zhu, Sebastian, Bachmann, David C, Dawson, David H, Ellison   +6 more
openaire   +2 more sources

Sweat chloride reflects CFTR function and correlates with clinical outcomes following CFTR modulator treatment.

Journal of Cystic Fibrosis
BACKGROUND Highly effective CFTR modulators improve CFTR function and lead to dramatic improvements in health outcomes in many people with cystic fibrosis (pwCF).
E. T. Zemanick, Bonnie W Ramsey, Dorota Sands, E. McKone, Isabelle Fajac, J. Taylor-Cousar, M. Mall, M. Konstan, N. Nair, Jiaqiang Zhu, E. Arteaga-Solis, F. van Goor, Lisa J. McGarry, Valentin Prieto-Centurion, P. Sosnay, Carmen Bozic, David Waltz, N. Mayer-Hamblett   +17 more
semanticscholar   +1 more source

CFTR Modulators: Current Status and Evolving Knowledge

Seminars in Respiratory and Critical Care Medicine, 2022
AbstractIn the past decade, the medical management of people with cystic fibrosis (pwCF) has changed with the development of small molecules that partially restore the function of the defective CF transmembrane conductance regulator (CFTR) protein and are called CFTR modulators.
Lucile, Regard, Clémence, Martin, Jennifer, Da Silva, Pierre-Régis, Burgel   +3 more
openaire   +2 more sources

CFTR Modulator Therapies for Cystic Fibrosis

Pediatric Allergy, Immunology, and Pulmonology, 2015
The cloning of cystic fibrosis transmembrane conductance regulator (CFTR) set into motion a cascade of discoveries that have helped to reveal the underlying pathophysiologic basis of cystic fibrosis (CF). This discovery and the knowledge that followed have also provided the opportunity to target this basic defect, with the hope of reversing or ...
Aaron T, Trimble, Scott H, Donaldson
openaire   +2 more sources