Results 51 to 60 of about 9,635 (207)

Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis

Health Technology Assessment, 2014
Background: Ivacaftor (Kalydeco®, Vertex Pharmaceuticals) is the first of a new class of drugs that target the underlying protein defect in cystic fibrosis (CF).
Penny Whiting, Maiwenn Al, Laura Burgers, Marie Westwood, Steve Ryder, Martine Hoogendoorn, Nigel Armstrong, Alex Allen, Hans Severens, Jos Kleijnen   +9 more
doaj   +1 more source

SLC26A9 Gene Is Associated With Lung Function Response to Ivacaftor in Patients With Cystic Fibrosis

Frontiers in Pharmacology, 2018
Ivacaftor is a drug used to treat cystic fibrosis (CF) patients carrying specific gating CFTR mutations. Interpatient variability in the lung response has been shown to be partly explained by rs7512462 in the Solute Carrier Family 26 Member 9 (SLC26A9 ...
Harriet Corvol, Harriet Corvol, Julie Mésinèle, Isman-Hassan Douksieh, Isman-Hassan Douksieh, Lisa J. Strug, Pierre-Yves Boëlle, Loïc Guillot   +7 more
doaj   +1 more source

Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes

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, Sophie Gohy, Michel P. Hermans, Vanessa Preumont   +3 more
doaj   +1 more source

Optimization of pharmaceutical research and development by early‐phase assessment of investigational medicinal products

British Journal of Clinical Pharmacology, EarlyView.
Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló, Attila Imre, Gábor Varga, Sándor Kovács, György Tímár, Balázs Nagy   +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, Chinedu Nwokoro, Mike McLean, Sarah Brown, Nanna Christiansen, Caroline S. Pao   +5 more
doaj   +1 more source

Advances in bronchiectasis:endotyping, genetics, microbiome, and disease heterogeneity [PDF]

, 2018
Bronchiectasis is characterised by pathological dilation of the airways. More specifically, the radiographic demonstration of airway enlargement is the common feature of a heterogeneous set of conditions and clinical presentations.
Chalmers, James D., Flume, Patrick A., Olivier, Kenneth N.   +2 more
core   +2 more sources

Long‐term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real‐world cohort study

British Journal of Clinical Pharmacology, EarlyView.
Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Nicola Perrotta, Luigi Angelo Fiorito, Roberta Vescovo, Anna Virgilio, Giulia Amato, Giuseppe Cimino   +5 more
wiley   +1 more source

G970R‐CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect

Clinical and Translational Science, 2021
In previous work, participants with a G970R mutation in cystic fibrosis transmembrane conductance regulator (CFTR) (c.2908G>C) had numerically lower sweat chloride responses during ivacaftor treatment than participants with other CFTR gating mutations ...
Meredith C. Fidler, Alexandra Buckley, James C. Sullivan, Marvin Statia, Sylvia F. Boj, Robert G. J. Vries, Anne Munck, Mark Higgins, Matteo Moretto Zita, Paul Negulescu, Fredrick vanGoor, Kris De Boeck   +11 more
doaj   +1 more source

Consistency and clarity of pharmacogenomic guidance in UK medicine patient information leaflets: A cross‐sectional analysis

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, Chloe Ratcliffe, Videha Sharma, William G. Newman, Munir Pirmohamed, John McDermott   +5 more
wiley   +1 more source

The effect of CFTR modulators on a cystic fibrosis patient presenting with recurrent pancreatitis in the absence of respiratory symptoms: a case report

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