Results 101 to 110 of about 1,046,440 (397)

VX‐659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

New England Journal of Medicine, 2018
BACKGROUND The next‐generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector VX‐659, in triple combination with tezacaftor and ivacaftor (VX‐659–tezacaftor–ivacaftor), was developed to restore the function of Phe508del CFTR protein
J. Davies, S. Moskowitz, C. Brown, A. Horsley, M. Mall, E. McKone, B. Plant, D. Prais, B. Ramsey, J. Taylor-Cousar, E. Tullis, A. Uluer, C. Mckee, S. Robertson, R. Shilling, C. Simard, F. van Goor, D. Waltz, F. Xuan, T. Young, S. Rowe   +20 more
semanticscholar   +1 more source

Designed Biomaterial‐Enhanced Cell Transplantation for Neural Tissue Engineering

Aggregate, EarlyView.
Biomaterials offer a promising solution for cell transplantation in the central nervous system by creating a protective environment that enhances cell survival, integration, and functional recovery in preclinical models of neurological disorders. ABSTRACT Cell transplantation therapy in the central nervous system is hindered by limited survival and ...
Yun Tang, Alice Le Friec, Menglin Chen, Di Sun   +3 more
wiley   +1 more source

Early respiratory viral infections in infants with cystic fibrosis [PDF]

, 2019
This article is made available for unrestricted research re-use and secondary analysis in any form or by any means with acknowledgement of the original source.
Davis, Stephanie D., Deschamp, Ashley R., Ferkol, Thomas W., Gebregziabher, Netsanet, Hall, Graham L., Hatch, Joseph E., Ranganathan, Sarath, Slaven, James E., Stick, Stephen, Storch, Gregory   +9 more
core   +2 more sources

Cystic fibrosis and the role of the physiotherapist

South African Journal of Physiotherapy, 1981
Cystic fibrosis is an inherited disease, the major problems being pancreatic malabsorption and progressive respiratory involvement, although numerous other organs may also be involved.
Anne Miot, J. M. Pettifor, I. Reef
doaj   +1 more source

Joint Hierarchical Gaussian Process Model with Application to Forecast in Medical Monitoring [PDF]

arXiv, 2014
A novel extrapolation method is proposed for longitudinal forecasting. A hierarchical Gaussian process model is used to combine nonlinear population change and individual memory of the past to make prediction. The prediction error is minimized through the hierarchical design.
arxiv  

New era of cystic fibrosis: full mutational analysis and personalized therapy [PDF]

, 2017
Despite its apparently simple genetics, cystic fibrosis (CF) is a rather complex genetic disease. A lot of variability in the steps of the path from the cystic fibrosis transmembrane conductance regulator (CFTR ) gene to the clinical manifestations ...
Lucarelli, Marco
core   +1 more source

Inflammation in cystic fibrosis: An update

Pediatric Pulmonology, 2018
Inflammation plays a critical role in cystic fibrosis (CF) lung pathology and disease progression making it an active area of research and important therapeutic target.
E. Roesch, D. Nichols, J. Chmiel
semanticscholar   +1 more source

Women's thoughts on receiving and sharing genetic information: Considerations for genetic counseling

Journal of Genetic Counseling, Volume 31, Issue 6, Page 1249-1260, December 2022., 2022
Abstract Indications for genetic testing for inherited cancer syndromes are expanding both in the academic and the community setting. However, only a fraction of individuals who are candidates for testing pursue this option. Therefore, it is important to understand those factors that impact the uptake of genetic testing in individuals affected and ...
Christopher D. Pfledderer, Lisa H. Gren, Caren J. Frost, Irene L. Andrulis, Wendy K. Chung, Jeanine Genkinger, Gord Glendon, John L. Hopper, Esther M. John, Melissa Southey, Mary Beth Terry, Mary B. Daly   +11 more
wiley   +1 more source

Ion mediated crosslink driven mucous swelling kinetics [PDF]

arXiv, 2015
We present an experimentally guided, multi-phasic, multi-species ionic gel model to compare and make qualitative predictions on the rheology of mucus of healthy individuals (Wild Type) versus those infected with Cystic Fibrosis. The mixture theory consists of the mucus (polymer phase) and water (solvent phase) as well as several different ions: H+, Na+
arxiv  

A factor graph EM algorithm for inference of kinetic microstates from patch clamp measurements [PDF]

arXiv, 2021
We derive a factor graph EM (FGEM) algorithm, a technique that permits combined parameter estimation and statistical inference, to determine hidden kinetic microstates from patch clamp measurements. Using the cystic fibrosis transmembrane conductance regulator (CFTR) and nicotinic acetylcholine receptor (nAChR) as examples, we perform {\em Monte Carlo}
arxiv