المؤلفون: Mayer-Hamblett, Nicole, Clancy, John Paul, Jain, Raksha, Donaldson, Scott H, Fajac, Isabelle, Goss, Christopher H, Polineni, Deepika, Ratjen, Felix, Quon, Bradley S, Zemanick, Edith T, Bell, Scott C, Davies, Jane C, Jain, Manu, Konstan, Michael W, Kerper, Natanya R, LaRosa, Tré, Mall, Marcus A, McKone, Edward, Pearson, Kelsie, Pilewski, Joseph M

المصدر: Lancet Respiratory Medicine; Oct2023, Vol. 11 Issue 10, p932-944, 13p

مصطلحات موضوعية: CYSTIC fibrosis transmembrane conductance regulator, CYSTIC fibrosis, NEW trials, CLINICAL trials

المؤلفون: Ramos, Kathleen J.¹ (AUTHOR) ramoskj@uw.edu, Pilewski, Joseph M.² (AUTHOR), Taylor-Cousar, Jennifer L.³ (AUTHOR)

المصدر: Journal of Cystic Fibrosis. May2021, Vol. 20 Issue 3, p381-387. 7p.

مصطلحات موضوعية: *CYSTIC fibrosis, *CYSTIC fibrosis transmembrane conductance regulator, *DIAGNOSIS, *LUNG transplantation, *LUNG diseases, *INTERSTITIAL lung diseases, *ATRIAL septal defects

الشركة/الكيان: UNITED States. Food & Drug Administration

المؤلفون: Horsley, Alexander, Donaldson, Scott H, Pilewski, Joseph M., Griese, Matthias, Cooke, Jon, Viswanathan, Lakshmi, Tullis, Elizabeth, Davies, Jane C., Lekstrom-Himes, Julie A., Wang, Linda T.

المصدر: Horsley, A, Donaldson, S H, Pilewski, J M, Griese, M, Cooke, J, Viswanathan, L, Tullis, E, Davies, J C, Lekstrom-Himes, J A & Wang, L T 2018, ' Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR ', American Journal of Respiratory and Critical Care Medicine, vol. 197, no. 2 . https://doi.org/10.1164/rccm.201704-0717OCTest

مصطلحات موضوعية: Male, 0301 basic medicine, Indoles, Cystic Fibrosis, Respiratory System, Administration, Oral, Cystic Fibrosis Transmembrane Conductance Regulator, Phases of clinical research, Quinolones, Pharmacology, Aminophenols, Critical Care and Intensive Care Medicine, Compound heterozygosity, Severity of Illness Index, Cystic fibrosis, Ivacaftor, 0302 clinical medicine, Medicine, Molecular Targeted Therapy, cystic fibrosis transmembrane conductance regulator corrector, biology, 11 Medical And Health Sciences, respiratory system, Prognosis, forced expiratory volume, Cystic fibrosis transmembrane conductance regulator, Respiratory Function Tests, Treatment Outcome, Drug Therapy, Combination, Female, sweat chloride, medicine.drug, Adult, Pulmonary and Respiratory Medicine, congenital, hereditary, and neonatal diseases and abnormalities, Adolescent, Maximum Tolerated Dose, Combination therapy, Risk Assessment, Drug Administration Schedule, Young Adult, 03 medical and health sciences, Pharmacotherapy, Double-Blind Method, Humans, Benzodioxoles, CFTR modulator, Dose-Response Relationship, Drug, business.industry, VX11-661-101 Study Group, Original Articles, medicine.disease, digestive system diseases, respiratory tract diseases, Regimen, 030104 developmental biology, 030228 respiratory system, biology.protein, business

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::39abdb03542155bf2ea5f41a6f10545cTest
https://www.research.manchester.ac.uk/portal/en/publications/tezacaftorivacaftor-in-subjects-with-cystic-fibrosis-and-f508delf508delcftr-or-f508delg551dcftrTest(99940aac-7bf9-40fc-8d3b-13f50b57efee).html

المؤلفون: Pilewski, Joseph M.¹ (AUTHOR) pilewskijm@upmc.edu, De Boeck, Kris² (AUTHOR), Nick, Jerry A.³ (AUTHOR), Tian, Simon⁴ (AUTHOR), DeSouza, Cynthia⁴ (AUTHOR), Higgins, Mark⁵ (AUTHOR), Moss, Richard B.⁶ (AUTHOR)

المصدر: Pulmonary Therapy. 2020, Vol. 6 Issue 2, p303-313. 11p.

مصطلحات موضوعية: *CYSTIC fibrosis transmembrane conductance regulator, *CYSTIC fibrosis

المؤلفون: Myerburg, Mike, Pilewski, Joseph M.

المصدر: American Journal of Respiratory & Critical Care Medicine; 7/1/2021, Vol. 204 Issue 1, p7-9, 3p

مصطلحات موضوعية: CYSTIC fibrosis treatment, CYSTIC fibrosis transmembrane conductance regulator, DRUG therapy, DRUG efficacy, LUNG transplantation, PHENOLS, HETEROCYCLIC compounds, CYSTIC fibrosis, MEMBRANE proteins

المؤلفون: Donaldson, Scott H.¹ (AUTHOR) scott_donaldson@med.unc.edu, Corcoran, Timothy E.² (AUTHOR), Pilewski, Joseph M.² (AUTHOR), Mogayzel, Peter³ (AUTHOR), Laube, Beth L.³ (AUTHOR), Boitet, Evan R.⁴ (AUTHOR), Harris, Elex S.⁴ (AUTHOR), Ceppe, Agathe¹ (AUTHOR), Edwards, Lloyd J.⁵ (AUTHOR), Zeman, Kirby¹ (AUTHOR), Wu, Jihong¹ (AUTHOR), Esther Jr, Charles R.⁶ (AUTHOR), Nichols, David P.⁷ (AUTHOR), Bennett, William D.¹ (AUTHOR), Rowe, Steven M.⁴ (AUTHOR)

المصدر: Journal of Cystic Fibrosis. Jan2024, Vol. 23 Issue 1, p155-160. 6p.

مصطلحات موضوعية: *MUCOCILIARY system, *CYSTIC fibrosis transmembrane conductance regulator, *CYSTIC fibrosis, *MUCUS

المؤلفون: Clancy, J P, Rowe, Steven M, Accurso, Frank J, Aitken, Moira L, Amin, Raouf S, Ashlock, Melissa A, Ballmann, Manfred, Boyle, Michael P, Bronsveld, Inez, Campbell, Preston W, De Boeck, Kris, Donaldson, Scott H, Dorkin, Henry L, Dunitz, Jordan M, Durie, Peter R, Jain, Manu, Leonard, Anissa, McCoy, Karen S, Moss, Richard B, Pilewski, Joseph M, Rosenbluth, Daniel B, Rubenstein, Ronald C, Schechter, Michael S, Botfield, Martyn, Ordoñez, Claudia L, Spencer-Green, George T, Vernillet, Laurent, Wisseh, Steve, Yen, Karl, Konstan, Michael W

المساهمون: UCL - (SLuc) Service de pédiatrie générale, UCL - SSS/IREC/PNEU - Pôle de Pneumologie, ORL et Dermatologie, UCL - (SLuc) Service de pneumologie

المصدر: Thorax : an international journal of respiratory medicine, Vol. 67, no. 1, p. 12-88 (2012)

مصطلحات موضوعية: Adolescent, Double-Blind Method, Female, Follow-Up Studies, Homozygote, Humans, Male, Middle Aged, Mutation, Prospective Studies, Sweat Glands, Adult, Treatment Outcome, Young Adult, Aminopyridines, Benzodioxoles, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, DNA, DNA Mutational Analysis, Dose-Response Relationship, Drug

العلاقة: boreal:126386; http://hdl.handle.net/2078.1/126386Test; info:pmid/21825083; urn:ISSN:0040-6376; urn:EISSN:1468-3296

الإتاحة: https://doi.org/10.1136/thoraxjnl-2011-200393Test
http://hdl.handle.net/2078.1/126386Test

المؤلفون: Hutt, Darren M, Herman, David, Rodrigues, Ana P C, Noël, Sabrina, Pilewski, Joseph M, Matteson, Jeanne, Hoch, Ben, Kellner, Wendy, Kelly, Jeffery W, Schmidt, Andre, Thomas, Philip J, Matsumura, Yoshihiro, Skach, William R, Gentzsch, Martina, Riordan, John R, Sorscher, Eric J, Okiyoneda, Tsukasa, Yates, John R, Lukacs, Gergely L, Frizzell, Raymond A, Manning, Gerard, Gottesfeld, Joel M, Balch, William E

المساهمون: UCL - Autre

المصدر: Nature Chemical Biology, Vol. 6, no.1, p. 25-33 (2010)

مصطلحات موضوعية: Animals, Humans, Hydroxamic Acids, Mutation, Protein Denaturation, Protein Folding, RNA, Small Interfering, Bronchi, Cell Membrane, Cricetinae, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Epithelial Cells, Gene Silencing, Histone Deacetylases

العلاقة: boreal:119719; http://hdl.handle.net/2078/119719Test; info:pmid/19966789; urn:ISSN:1552-4450; urn:EISSN:1552-4469

الإتاحة: https://doi.org/10.1038/nchembio.275Test
http://hdl.handle.net/2078/119719Test

المؤلفون: Accurso, Frank J., Rowe, Steven M., Clancy, J.P., Boyle, Michael P., Dunitz, Jordan M., Durie, Peter R., Sagel, Scott D., Hornick, Douglas B., Konstan, Michael W., Donaldson, Scott H., Moss, Richard B., Pilewski, Joseph M., Rubenstein, Ronald C., Uluer, Ahmet Z., Aitken, Moira L., Freedman, Steven D., Rose, Lynn M., Mayer-Hamblett, Nicole, Dong, Qunming, Zha, Jiuhong, Stone, Anne J., Olson, Eric R., Ordoñez, Claudia L., Campbell, Preston W., Ashlock, Melissa A., Ramsey, Bonnie W.

المصدر: The New England Journal of Medicine, 363(21)

مصطلحات موضوعية: Metabolic diseases, Mutación, Pathologie du pancréas, Mutation, Pathologie de l'appareil digestif, Pancreatic disease, Cystic fibrosis transmembrane conductance regulator, Pathologie de l'appareil respiratoire, Respiratory disease, Genetics, Digestive diseases, Génétique, Maladie héréditaire, Mucoviscidose, Médecine, Medicina, Regulator conductancia transmembrana mucoviscidosis, Régulateur conductance transmembranaire mucoviscidose, Medicine, Maladie métabolique, Genética, Genetic disease, Cystic fibrosis

العلاقة: https://doi.org/10.17615/bbpq-c156Test; https://cdr.lib.unc.edu/downloads/m900p305p?file=thumbnailTest; https://cdr.lib.unc.edu/downloads/m900p305pTest

الإتاحة: https://doi.org/10.17615/bbpq-c156Test
https://cdr.lib.unc.edu/downloads/m900p305p?file=thumbnailTest
https://cdr.lib.unc.edu/downloads/m900p305pTest

المؤلفون: Bertrand, Carol A., Mitra, Shalini, Mishra, Sanjay K., Xiaohui Wang, Yu Zhao, Pilewski, Joseph M., Madden, Dean R., Frizzell, Raymond A.

المصدر: American Journal of Physiology: Lung Cellular & Molecular Physiology; Jun2017, Vol. 312 Issue 6, p912-925, 14p

مصطلحات موضوعية: CYSTIC fibrosis transmembrane conductance regulator, CHLORIDE channels, GENETIC mutation