Results 21 to 30 of about 1,122,604 (303)

Targeted therapies to improve CFTR function in cystic fibrosis [PDF]

, 2015
Cystic fibrosis is the most common genetically determined, life-limiting disorder in populations of European ancestry. The genetic basis of cystic fibrosis is well established to be mutations in the cystic fibrosis transmembrane conductance regulator ...
Brodlie, M, Elborn, JS, Haq, IJ, Roberts, K   +3 more
core   +2 more sources

T helper cell subsets specific for pseudomonas aeruginosa in healthy individuals and patients with cystic fibrosis [PDF]

, 2014
Background: We set out to determine the magnitude of antigen-specific memory T helper cell responses to Pseudomonas aeruginosa in healthy humans and patients with cystic fibrosis.
AJ Vallis, AL Gurney, B Stockinger, BJ Stoodley, CE Zielinski, DG Downey, Dominik Hartl, EV Acosta-Rodriguez, F Ratjen, GF Sonnenberg, GF Sonnenberg, Gordon MacGregor, GP Priebe, Hannah K. Bayes, HL Tan, K Chen, K Wolk, L Martinez-Solano, M Brodlie, M Wuthrich, N Ota, N Rieber, PJ Dubin, R Basu, RB Markham, RG Wunderink, RT Sadikot, S Eyerich, S Trifari, SJ Aujla, SL Gaffen, SM Rowe, Stephen Bicknell, T Duhen, T Sawa, Tom J. Evans, Y Zheng   +36 more
core   +3 more sources

Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.

New England Journal of Medicine, 2019
BACKGROUND Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del CFTR mutation.
P. Middleton, M. Mall, P. Dřevínek, L. Lands, E. McKone, Deepika Polineni, B. Ramsey, J. Taylor-Cousar, E. Tullis, F. Vermeulen, G. Marigowda, C. Mckee, S. Moskowitz, N. Nair, J. Savage, C. Simard, S. Tian, D. Waltz, F. Xuan, S. Rowe, R. Jain   +20 more
semanticscholar   +1 more source

Virtual Peer Support for People With Cystic Fibrosis and Their Family Members: A Program Evaluation

Journal of Patient Experience, 2020
We sought to evaluate the feasibility, acceptability, and benefits of a virtual one-to-one peer support program for people with cystic fibrosis and their family members through a retrospective program evaluation.
Aimee Jeffrey MSN, Lauren Andracchio MPH, Meg Dvorak LCSW, Paula Lomas MAS, RN, CCRP, Beth Smith MD, Drucy Borowitz MD   +5 more
doaj   +1 more source

The impact of 12 months treatment with ivacaftor on Scottish paediatric patients with cystic fibrosis with the G551D mutation : a review [PDF]

, 2016
We reviewed the impact of ivacaftor on Scottish paediatric cystic fibrosis (CF) patients ≥ 6 years of age after 12 months of treatment. Statistically significant improvements in FEV1 and BMI and a reduction in sweat chloride, all comparable with ...
Scottish Paediatric Cystic Fibrosis Managed Clinical Network (SPCFMCN)
core   +1 more source

VX-659–Tezacaftor–Ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles

Canadian Journal of Respiratory Critical Care and Sleep Medicine, 2020
Cystic fibrosis is an autosomal-recessive, multisystem disease that affects ∼80,000 patients worldwide.1,2 It is caused by mutations in the CFTR gene, an epithelial chloride ion channel with varyin...
E. Merman, M. Stanbrook, A. Anand
semanticscholar   +1 more source

Cystic fibrosis mice carrying the missense mutation G551D replicate human genotype phenotype correlations [PDF]

, 1996
We have generated a mouse carrying the human G551D mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR) by a one-step gene targeting procedure.
Alton, Ewfw, Delaney, S. J., Dorin, J. R., Farley, R., Hume, D. A., Lamb, D., Lovelock, P. K., Lunn, D. P., Porteous, D. J., Smith, S. N., Thomson, S. A., Wainwright, B. J.   +11 more
core   +2 more sources

Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity

JAMA Network Open, 2020
This cross-sectional study compares disease-specific federal and foundation funding for sickle cell disease and cystic fibrosis research and assesses the factors associated with research productivity.
Faheem Farooq, P. Mogayzel, S. Lanzkron, C. Haywood, J. Strouse   +4 more
semanticscholar   +1 more source

Cystic fibrosis

Jornal de Pediatria, 1998
OBJECTIVE: Due to the great advances recently achieved in the treatment of Cystic Fibrosis as well as to the fact that pediatricians need to have a better understanding of this disease, the authors propose an extensive review of the subject. METHODS: We selected the most outstanding publications on Cystic Fibrosis in the international literature of the
F J, Reis, N, Damaceno
openaire   +4 more sources

Cystic Fibrosis Foundation and European Cystic Fibrosis Society Survey of cystic fibrosis mental health care delivery [PDF]

, 2015
Background: Psychological morbidity in individuals with cystic fibrosis (CF) and their caregivers is common. The Cystic Fibrosis Foundation (CFF) and European Cystic Fibrosis Society (ECFS) Guidelines Committee on Mental Health sought the views of CF ...
A.L. Quittner, A.M. Georgiopoulos, B.A. Smith, B.C. Marshall, Boyle, Braun, Clarke, DiMatteo, Fidika, Foy, Foy, Godfrey, Havermans, Health Disorders, J. Abbott, J.S. Elborn, K.A. Sabadosa, L. Goldbeck, Lewiston, Manfreda, Mitchell, Monkey, Moussavi, National Collaborating Centre For Mental Health (UK), National Collaborating Centre for Mental Health (UK), Nulty, Quittner, Riekert, Sawicki, Shih, Smith, Snell, Stern, West   +33 more
core   +1 more source