Results 51 to 60 of about 9,731 (210)
British Journal of Clinical Pharmacology, EarlyView.Pharmacogenomic (PGx) information has the potential to support the safe and effective use of medicines, yet there is uncertainty about how this information can be best communicated to patients. Summaries of product characteristics (SmPCs) and patient information leaflets (PILs) for all UK‐approved medicines with strong evidence supporting a PGx ...
Parth Narlawar +5 more
wiley +1 more source
Real-world effectiveness of ivacaftor in children with cystic fibrosis and the G551D mutation
Anales de Pediatría (English Edition), 2019 Introduction: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has been shown to improve the nutritional status and lung function of cystic fibrosis patients with the G551D mutation in clinical trials.
David Gomez-Pastrana +5 more
doaj +1 more source
Cystic fibrosis modulator therapy can reverse cystic bronchiectasis
Respirology Case Reports, 2023 Bronchiectasis is often considered progressive and irreversible, so cases of regression or reversal are an important step in understanding the underlying pathophysiological mechanisms.
Peter G. Middleton, Nicholas J. Simmonds
doaj +1 more source
Journal of Clinical & Translational Endocrinology, 2023 Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin +3 more
doaj +1 more source
Newborn screening for cystic fibrosis [PDF]
, 2016 Since the late 1970s when the potential of the immunoreactive trypsinogen assay for early identification of infants with cystic fibrosis was first recognised, the performance of newborn blood spot screening (NBS) has been continually assessed and its use
Castellani, C +3 more
core +1 more source
Journal of Magnetic Resonance Imaging, EarlyView.ABSTRACT Background Elexacaftor/tezacaftor/ivacaftor (ETI) is a current standard therapy for pediatric cystic fibrosis (CF). Multiple‐breath washout 129Xe MRI (MBW Xe‐MRI) is improved following 1 month of treatment. However, the utility of MBW Xe‐MRI over extended ETI treatment and its comparison to single‐breath Xe‐MRI and pulmonary function tests ...
Faiyza S. Alam +6 more
wiley +1 more source
Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants. [PDF]
, 2019 Available CFTR modulators provide no therapeutic benefit for cystic fibrosis (CF) caused by many loss-of-function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel, including N1303K. We previously introduced the
Finkbeiner, Walter E +7 more
core
The use of Multiple Breath Washout for Assessing Cystic Fibrosis in Infants [PDF]
, 2016 INTRODUCTION: Lung Clearance Index, measured using the multiple breath washout (MBW) technique, may be a useful test in infants with Cystic Fibrosis (CF).
Aurora, P, Davies, G
core +1 more source
JPGN Reports, EarlyView.Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano +12 more
wiley +1 more source
BMC Gastroenterology, 2019 Background Cystic fibrosis (CF) is a genetic disorder of the epithelial CFTR apical chloride channel resulting in multi-organ manifestations, including pancreatic exocrine secretion.
J. Dixon Johns, Steven M. Rowe
doaj +1 more source