المؤلفون: A. R. Kosareva, O. A. Bashkina, D. F. Sergienko

المصدر: Acta Biomedica Scientifica, Vol 7, Iss 4, Pp 101-108 (2022)

مصطلحات موضوعية: cystic fibrosis, orkambi, lumacaftor/ivacaftor, children, astrakhan region, Science

وصف الملف: electronic resource

Relation: https://www.actabiomedica.ru/jour/article/view/3650; https://doaj.org/toc/2541-9420; https://doaj.org/toc/2587-9596

URL الوصول: https://doaj.org/article/610044a4288f48b5a616144f5f4b27aa

المؤلفون: Melissa Iazzi, Audrey Astori, Jonathan St-Germain, Brian Raught, Gagan Gupta

مصطلحات موضوعية: Cell biology, n.e.c, CFTR interactions, CFTR Modulators, cystic fibrosis, Orkambi, theratyping, protein trafficking, chaperones, transmembrane conductance regulator, protein-quality control, cystic-fibrosis, endoplasmic-reticulum, affinity purification, lumacaftor-ivacaftor, ER, identification, biotinylation, degradation

Relation: https://figshare.com/articles/journal_contribution/Proximity_Profiling_of_the_CFTR_Interaction_Landscape_in_Response_to_Orkambi/21948443

الاتاحة: https://doi.org/10.32920/21948443.v1
https://figshare.com/articles/journal_contribution/Proximity_Profiling_of_the_CFTR_Interaction_Landscape_in_Response_to_Orkambi/21948443

المؤلفون: Melissa Iazzi, Audrey Astori, Jonathan St-Germain, Brian Raught, Gagan D. Gupta

المصدر: International Journal of Molecular Sciences; Volume 23; Issue 5; Pages: 2442

مصطلحات موضوعية: CFTR interactions, CFTR modulators, cystic fibrosis, Orkambi, theratyping, protein trafficking, chaperones

جغرافية الموضوع: agris

وصف الملف: application/pdf

Relation: Molecular Pathology, Diagnostics, and Therapeutics; https://dx.doi.org/10.3390/ijms23052442

الاتاحة: https://doi.org/10.3390/ijms23052442

المؤلفون: Moran, Gillian, author, Mountford, Nicola, author

المصدر: Healthcare Activism : Markets, Morals, and the Collective Good, 2021, ill.

الاتاحة: https://doi.org/10.1093/oso/9780198865223.003.0004

المؤلفون: Connett GJ

المصدر: Drug Design, Development and Therapy, Vol Volume 13, Pp 2405-2412 (2019)

مصطلحات موضوعية: lumacaftor, ivacaftor, orkambi, modulators, correctors, potentiators, cystic fibrosis, CFTR, Therapeutics. Pharmacology, RM1-950

وصف الملف: electronic resource

Relation: https://www.dovepress.com/lumacaftor-ivacaftor-in-the-treatment-of-cystic-fibrosis-design-develo-peer-reviewed-article-DDDT; https://doaj.org/toc/1177-8881

URL الوصول: https://doaj.org/article/9c869993f6bf4a1dab3bd0454b78e917

المؤلفون: Falchetti, Mélanie

المساهمون: Université de Bordeaux (UB), Jean-François Quignard

المصدر: https://dumas.ccsd.cnrs.fr/dumas-04513614 ; Sciences du Vivant [q-bio]. 2024.

مصطلحات موضوعية: Cystic fibrosis, Genetics, CFTR channel, Lungs, Pancreas, Protein therapy, CFTR channel modulators, Kalydeco®, Orkambi®, Symkevi®, Kaftrio®, Potentiator, Corrector, Mucoviscidose, Génétique, Canal CFTR, Poumons, Pancréas, Thérapie protéique, Modulateurs du canal CFTR, Potentiateur, Correcteur, [SDV]Life Sciences [q-bio], [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology, [SDV.MHEP.CHI]Life Sciences [q-bio]/Human health and pathology/Surgery

Relation: dumas-04513614; https://dumas.ccsd.cnrs.fr/dumas-04513614; https://dumas.ccsd.cnrs.fr/dumas-04513614/document; https://dumas.ccsd.cnrs.fr/dumas-04513614/file/Pharmacie_2024_Falchetti.pdf

الاتاحة: https://dumas.ccsd.cnrs.fr/dumas-04513614
https://dumas.ccsd.cnrs.fr/dumas-04513614/document
https://dumas.ccsd.cnrs.fr/dumas-04513614/file/Pharmacie_2024_Falchetti.pdf

المؤلفون: Favia M., Gallo C., Guerra L., De Venuto D., Diana A., Polizzi A. M., Montemurro P., Mariggio M. A., Leonetti G., Manca A., Casavola V., Conese M.

المساهمون: Favia, M., Gallo, C., Guerra, L., De Venuto, D., Diana, A., Polizzi, A. M., Montemurro, P., Mariggio, M. A., Leonetti, G., Manca, A., Casavola, V., Conese, M.

مصطلحات موضوعية: Cystic Fibrosi, Orkambi, CFTR ,mononuclear cells, BMI, sweat chloride, FEV1

Relation: info:eu-repo/semantics/altIdentifier/pmid/32244302; info:eu-repo/semantics/altIdentifier/wos/WOS:000535574200140; volume:21; issue:7; firstpage:2398; journal:INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES; https://hdl.handle.net/11586/295252

الاتاحة: https://hdl.handle.net/11586/295252
https://doi.org/10.3390/ijms21072398

المؤلفون: Gamet, Fanny

المساهمون: Aix-Marseille Université - Faculté de pharmacie (AMU PHARM), Aix Marseille Université (AMU), Camille Desgrouas

المصدر: Sciences pharmaceutiques. 2023

مصطلحات موضوعية: SYMKEVI ®, KAFTRIO ®, [SDV]Life Sciences [q-bio], Gène CFTR, [SDV.SP]Life Sciences [q-bio]/Pharmaceutical sciences, ORKAMBI ®, Maladies rares, Plan France Médecine Génomique 2025, Médicaments orphelins, Désignation orpheline, Mesures incitatives, Mucoviscidose, Plans nationaux, Mutations, KALYDECO ®

URL الوصول: https://explore.openaire.eu/search/publication?articleId=od______3430::832198664c7041abb99ebe69ec3500d9
https://dumas.ccsd.cnrs.fr/dumas-04061185/document

المؤلفون: Elizabeth Matthes, Julie Goepp, Carolina Martini, Jiajie Shan, Jie Liao, David Y. Thomas, John W. Hanrahan

المصدر: Frontiers in Pharmacology, Vol 9 (2018)

مصطلحات موضوعية: precision medicine, cystic fibrosis, correctors, lumacaftor, Orkambi, group sampling, Therapeutics. Pharmacology, RM1-950

وصف الملف: electronic resource

Relation: https://www.frontiersin.org/article/10.3389/fphar.2018.01490/full; https://doaj.org/toc/1663-9812

URL الوصول: https://doaj.org/article/1125c634de6d439eb5cd4b9a631d19ee

المؤلفون: A. S. Efremova, T. B. Bukharova, N. Y. Kashirskaya, D. V. Goldshtein, А. С. Ефремова, Т. Б. Бухарова, Н. Ю. Каширская, Д. В. Гольдштейн

المصدر: Medical Genetics; Том 17, № 9 (2018); 3-12 ; Медицинская генетика; Том 17, № 9 (2018); 3-12 ; 2073-7998

مصطلحات موضوعية: forskolin-induced swelling assay, кишечные органоиды, муковисцидоз, CFTR-таргетные препараты, калидеко, оркамби, форсколиновый тест, organoids, intestinal organoids, cystic fibrosis, CFTR-targeting drugs, «Kalydeco», «Orkambi»

وصف الملف: application/pdf

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Helicobacter-induced gastric inflammation alters the properties of gastric tissue stem/progenitor cells. BMC Gastroenterol. 2017;17(1).; Hohwieler M, Illing A, Hermann PC, et al. Human pluripotent stem cell-derived acinar/ductal organoids generate human pancreas upon orthotopic transplantation and allow disease modelling. Gut. 2017;66(3):473-486.; Takasato M, Er PX, Chiu HS, et al. Kidney organoids from human iPS cells contain multiple lineages and model human nephrogenesis. Nature. 2015;526(7574):564-568.; Garreta E, Gonzalez F, Montserrat N. Studying kidney disease using tissue and genome engineering in human pluripotent stem cells. Nephron. 2018;138(1):48-59.; Fujii M, Shimokawa M, Date S, et al. A colorectal tumor organoid library demonstrates progressive loss of niche factor requirements during tumorigenesis. Cell Stem Cell. 2016;18(6):827-838.; Buzzelli JN, Ouaret D, Brown G, et al. Colorectal cancer liver metastases organoids retain characteristics of original tumor and acquire chemotherapy resistance. Stem Cell Res. 2018;27:109-120.; Boehnke K, Iversen PW, Schumacher D, et al. Assay establishment and validation of a high-throughput screening platform for three-dimensional patient-derived colon cancer organoid cultures. J Biomol Screen. 2016;21(9):931-941.; Pauli C, Hopkins BD, Prandi D, et al. Personalized In Vitro and In Vivo Cancer Models to Guide Precision Medicine. Cancer Discov. 2017;7(5):462-477.; Hill DR, Huang S, Nagy MS, et al. Bacterial colonization stimulates a complex physiological response in the immature human intestinal epithelium. Elife. 2017;6.; Ley S, Galuba O, Salathe A, et al. Screening of intestinal crypt organoids: a simple readout for complex biology. SLAS Discov. 2017;22(5):571-582.; Schwank G, Koo BK, Sasselli V, et al. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. Cell Stem Cell. 2013;13(6):653-658.; Cocola C, Molgora S, Piscitelli E, et al. FGF2 and EGF are required for self-renewal and organoid formation of canine normal and tumor breast stem cells. J Cell Biochem. 2017;118(3):570-584.; Takahashi Y, Sato S, Kurashima Y, et al. A refined culture system for human induced pluripotent stem cell-derived intestinal epithelial organoids. Stem Cell Reports. 2018;10(1):314-328.; Hohwieler M, Perkhofer L, Liebau S, et al. Stem cell-derived organoids to model gastrointestinal facets of cystic fibrosis. United European Gastroenterol J. 2017;5(5):609-624.; Balimane PV, Chong S. Cell culture-based models for intestinal permeability: a critique. Drug Discov Today. 2005;10(5):335-343.; Sato T, Stange DE, Ferrante M, et al. Long-term expansion of epithelial organoids from human colon, adenoma, adenocarcinoma, and Barrett’s epithelium. Gastroenterology. 2011;141(5):1762-1772.; Sato T, Clevers H. Growing self-organizing mini-guts from a single intestinal stem cell: mechanism and applications. Science. 2013;340(6137):1190-1194.; Meneses AM, Schneeberger K, Kruitwagen HS, et al. Intestinal organoids - current and future applications. Vet Sci. 2016;3(4).; Beekman JM. Individualized medicine using intestinal responses to CFTR potentiators and correctors. Pediatr Pulmonol. 2016;51(S44):S23-S34.; Noordhoek J, Gulmans V, van der Ent K, et al. Intestinal organoids and personalized medicine in cystic fibrosis: a successful patient-oriented research collaboration. Curr Opin Pulm Med. 2016;22(6):610-616.; Dekkers JF, van der Ent CK, Beekman JM. Novel opportunities for CFTR-targeting drug development using organoids. Rare Dis. 2013;1(e27112).; Matsuzawa-Ishimoto Y, Shono Y, Gomez LE, et al. Autophagy protein ATG16L1 prevents necroptosis in the intestinal epithelium. J Exp Med. 2017;214(12):3687-3705.; Watson CL, Mahe MM, Munera J, et al. An in vivo model of human small intestine using pluripotent stem cells. Nat Med. 2014;20(11):1310-1314.; Смирнихина СА, Банников АВ, Лавров АВ. Оптимизация условий трансфекции клеточной культуры CFTE29o- для разработки редактирования мутации F508del в гене CFTR. Медицинская генетика. 2016;8:36-39.; Смирнихина СА, Банников АВ, Анучина АА и др. Факторы, влияющие на эффективность CRISPR/Cas9 для коррекции мутации F508del при муковисцидозе. Медицинская генетика. 2017;11:32-37.; Okkelman IA, Foley T, Papkovsky DB, et al. Multi-parametric imaging of hypoxia and cell cycle in intestinal organoid culture. Adv Exp Med Biol. 2017;1035:85-103.; Kraiczy J, Nayak KM, Howell KJ, et al. DNA methylation defines regional identity of human intestinal epithelial organoids and undergoes dynamic changes during development. Gut. 2017;0:1-13.; Thalheim T, Quaas M, Herberg M, et al. Linking stem cell function and growth pattern of intestinal organoids. Dev Biol. 2018;433(2):254-261.; Bartfeld S, Clevers H. Stem cell-derived organoids and their application for medical research and patient treatment. J Mol Med (Berl). 2017;95(7):729-738.; Carmon KS, Gong X, Lin Q, et al. R-spondins function as ligands of the orphan receptors LGR4 and LGR5 to regulate Wnt/beta-catenin signaling. Proc Natl Acad Sci U S A. 2011;108(28):11452-11457.; Dekkers JF, Berkers G, Kruisselbrink E, et al. Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis. Sci Transl Med. 2016;8(344):344ra384.; Cruz-Acuna R, Quiros M, Farkas AE, et al. Synthetic hydrogels for human intestinal organoid generation and colonic wound repair. Nat Cell Biol. 2017;19(11):1326-1335.; Boj SF, Vonk AM, Statia M, et al. Forskolin-induced swelling in intestinal organoids: an in vitro assay for assessing drug response in cystic fibrosis patients. J Vis Exp. 2017;120.; Муковисцидоз (ред. Капранов НИ, Каширская НЮ). М: Медпрактика-М, 2014:672 с.; Quon BS, Rowe SM. New and emerging targeted therapies for cystic fibrosis. BMJ. 2016;352(i859).; Hurley MN, McKeever TM, Prayle AP, et al. Rate of improvement of CF life expectancy exceeds that of general population-observational death registration study. J Cyst Fibros. 2014;13(4):410-415.; https://www.healthline.com/health/cystic-fibrosis-facts; Sosnay PR, Siklosi KR, Van Goor F, et al. Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene. Nat Genet. 2013;45(10):1160-1167.; Quintana-Gallego E, Delgado-Pecellin I, Calero Acuna C. CFTR protein repair therapy in cystic fibrosis. Arch Bronconeumol. 2014;50(4):146-150.; Петрова НВ, Кондратьева ЕИ, Красовский СА и др. Проект национального консенсуса «Муковисцидоз: определение, диагностические критерии, терапия» Раздел «Генетика муковисцидоза. Молекулярно-генетическая диагностика при муковисцидозе». Медицинская генетика. 2016;15(11):29-45.; Welsh MJ, Smith AE. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell. 1993;73(7):1251-1254.; Zielenski J, Tsui LC. Cystic fibrosis: genotypic and phenotypic variations. Annu Rev Genet. 1995;29:777-807.; Van Goor F, Hadida S, Grootenhuis PD, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci U S A. 2009;106(44):18825-18830.; Регистр больных муковисцидозом в Российской Федерации. 2016 год. (ред. Красовский СА, Черняк АВ, Воронкова АЮ, Амелина ЕЛ, Каширская НЮ, Кондратьева ЕИ, Гембицкая ТЕ). М: Медпрактика-М, 2018:64 с.; De Boeck K, Wilschanski M, Castellani C, et al. Cystic fibrosis: terminology and diagnostic algorithms. Thorax. 2006;61(7):627-635.; Van Goor F, Straley KS, Cao D, et al. Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am J Physiol Lung Cell Mol Physiol. 2006;290(6):L1117-1130.; Maiuri L, Raia V, Kroemer G. Strategies for the etiological therapy of cystic fibrosis. Cell Death Differ. 2017;24(11):1825-1844.; Zainal Abidin N, Haq IJ, Gardner AI, et al. Ataluren in cystic fibrosis: development, clinical studies and where are we now? Expert Opin Pharmacother. 2017;18(13):1363-1371.; Pedemonte N, Lukacs GL, Du K, et al. Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening. J Clin Invest. 2005;115(9):2564-2571.; Accurso FJ, Rowe SM, Durie PR, et al. Interim results of Phase IIa study of VX-770 to evaluate safety, pharmokinetics and biomarkers of CFTR activity in cystic fibrosis subjects with G551D. Pediatr Pulmonol. 2008;Suppl 31:267-295.; Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672.; https://www.cff.org/Life-With-CF/Treatments-and-Therapies/CFTR-Modulator-Therapies/; Taylor-Cousar JL, Munck A, McKone EF, et al. Tezacaftor-Ivacaftor in patients with cystic fibrosis homozygous for Phe508del. N Engl J Med. 2017;377(21):2013-2023.; Saini A. Cystic ffibrosis patients benefit from mini guts. Cell Stem Cell. 2016;19:425-427.; Dekkers R, Vijftigschild LA, Vonk AM, et al. A bioassay using intestinal organoids to measure CFTR modulators in human plasma. J Cyst Fibros. 2015;14(2):178-181.; https://www.medgen-journal.ru/jour/article/view/579

الاتاحة: https://www.medgen-journal.ru/jour/article/view/579

المؤلفون: G J, Connett

المصدر: Drug Design, Development and Therapy, Vol Volume 13, Pp 2405-2412 (2019)
Drug Design, Development and Therapy

مصطلحات موضوعية: modifier, lumacaftor, lcsh:RM1-950, Phe508del, Aminopyridines, Cystic Fibrosis Transmembrane Conductance Regulator, modulator, Review, Quinolones, Aminophenols, orkambi, cystic fibrosis, Drug Combinations, correctors, lcsh:Therapeutics. Pharmacology, Drug Design, Humans, ivacaftor, Drug Therapy, Combination, modulators, Benzodioxoles, potentiators, CFTR, corrector, DF508

URL الوصول: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::49eef9e9a1f75ae3a44bd2841eeb8258
https://www.dovepress.com/lumacaftor-ivacaftor-in-the-treatment-of-cystic-fibrosis-design-develo-peer-reviewed-article-DDDT

المؤلفون: Španić, Tena

المساهمون: Banac, Srđan, Palčevski, Goran, Bulat-Kardum, Ljiljana, Vitezić, Dinko

مصطلحات موضوعية: korektor, potencijator, Symdeko, Trikafta, cistična fibroza, BIOMEDICINE AND HEALTHCARE. Clinical Medical Sciences. Pediatrics, Kalydeko, Orkambi, BIOMEDICINA I ZDRAVSTVO. Kliničke medicinske znanosti. Pedijatrija

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::2cabc72f13aea84eefccf9a3814c6502
https://repository.medri.uniri.hr/islandora/object/medri:4633/datastream/PDF

المؤلفون: Abdulsabar Sanni, Anas Zaher, Jude ElSaygh, Hassan ElSaygh, Dalal H Elsori

المصدر: Cureus

مصطلحات موضوعية: elexacaftor, Pulmonology, trikafta, Disease, Bioinformatics, Pediatrics, orkambi, Cystic fibrosis, cystic fibrosis, Ivacaftor, chemistry.chemical_compound, Internal Medicine, medicine, Adverse effect, cystic fibrosis therapy, Lung, biology, lumacaftor, business.industry, Lumacaftor, General Engineering, cystic fibrosis triple therapy, medicine.disease, Cystic fibrosis transmembrane conductance regulator, Clinical trial, medicine.anatomical_structure, chemistry, tezacaftor, biology.protein, ivacaftor, business, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b7aff5a480c5a6e010a42a81e34eb734
https://doi.org/10.7759/cureus.16144

المؤلفون: Sarah AlBabtain, Mehwish Zeb, Muhammad Reza Chaudhry, Sana Amreen, Safeera Khan, Sumera Perveen, Simrandeep K Brar

المصدر: Cureus

مصطلحات موضوعية: medicine.medical_specialty, Disease, 030204 cardiovascular system & hematology, Cystic fibrosis, Pediatrics, orkambi, Ivacaftor, cystic fibrosis, ivacaftor/lumacaftor combination, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Quality of life, Internal medicine, medicine, Internal Medicine, Disease burden, cystic fibrosis/therapy, biology, business.industry, lumacaftor, Lumacaftor, General Engineering, medicine.disease, Cystic fibrosis transmembrane conductance regulator, Clinical trial, chemistry, biology.protein, ivacaftor, cystic fibrosis/drug therapy, business, 030217 neurology & neurosurgery, Family/General Practice, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d25520c426c1cbff0a4dc42304f2a95f
http://europepmc.org/articles/PMC7834585

المؤلفون: Lorenzo Guerra, Anna Diana, Giuseppina Leonetti, Angela Polizzi, Massimo Conese, Maria Addolorata Mariggiò, Valeria Casavola, Maria Favia, Antonio Manca, Domenica De Venuto, Pasqualina Montemurro, Crescenzio Gallo

المصدر: International Journal of Molecular Sciences, Vol 21, Iss 2398, p 2398 (2020)
International Journal of Molecular Sciences
Volume 21
Issue 7

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, congenital, hereditary, and neonatal diseases and abnormalities, chemical and pharmacologic phenomena, FEV1%, Peripheral blood mononuclear cell, Cystic fibrosis, Catalysis, Inorganic Chemistry, Ivacaftor, cystic fibrosis, lcsh:Chemistry, 03 medical and health sciences, chemistry.chemical_compound, BMI, 0302 clinical medicine, immune system diseases, White blood cell, Internal medicine, hemic and lymphatic diseases, medicine, Respiratory function, Physical and Theoretical Chemistry, CFTR, Molecular Biology, lcsh:QH301-705.5, Spectroscopy, mononuclear cells, Orkambi®, business.industry, Organic Chemistry, Lumacaftor, hemic and immune systems, General Medicine, Potentiator, medicine.disease, Computer Science Applications, 030104 developmental biology, medicine.anatomical_structure, Endocrinology, chemistry, lcsh:Biology (General), lcsh:QD1-999, 030220 oncology & carcinogenesis, business, sweat chloride, Ex vivo, medicine.drug

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8d1b2b50320e5c665f872001b4ef792b
https://www.mdpi.com/1422-0067/21/7/2398

المؤلفون: Maria, Favia, Crescenzio, Gallo, Lorenzo, Guerra, Domenica, De Venuto, Anna, Diana, Angela Maria, Polizzi, Pasqualina, Montemurro, Maria Addolorata, Mariggiò, Giuseppina, Leonetti, Antonio, Manca, Valeria, Casavola, Massimo, Conese

المصدر: International Journal of Molecular Sciences

مصطلحات موضوعية: Adult, Male, Adolescent, Cystic Fibrosis, Patients, Aminopyridines, Cystic Fibrosis Transmembrane Conductance Regulator, Orkambi®, Quinolones, FEV1%, Aminophenols, Article, Body Mass Index, Leukocyte Count, Young Adult, BMI, Chlorides, Forced Expiratory Volume, Leukocytes, Humans, Benzodioxoles, CFTR, Child, mononuclear cells, Homozygote, Middle Aged, Respiratory Function Tests, Drug Combinations, Mutation, Female, sweat chloride

URL الوصول: https://explore.openaire.eu/search/publication?articleId=pmid________::aa9439f92028361ef048975d12d99573
https://pubmed.ncbi.nlm.nih.gov/32244302

المؤلفون: Arends AM, Pettit RS

المصدر: Orphan Drugs: Research and Reviews, Vol 2015, Iss Issue 1, Pp 61-68 (2015)

مصطلحات موضوعية: cystic fibrosis, lcsh:Therapeutics. Pharmacology, lumacaftor, lcsh:RM1-950, ivacaftor, VX-809, VX-770, orkambi

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doajarticles::0d49835eed90bd52b04716899563955a
https://www.dovepress.com/profile-of-lumacaftorivacaftor-combination-potential-in-the-treatment--peer-reviewed-article-ODRR

المؤلفون: Aparicio Hernández, R., García Luque, A., Gonzalo Salado, M.ªH., Cabanes Mariscal, M.A.

المصدر: Sanidad Militar, Volume: 72, Issue: 1, Pages: 42-52, Published: MAR 2016

مصطلحات موضوعية: Cotellic®, Ravicti®, Blinatumomab, Genvoya®, Idarucizumab, Pegaspargasa, Orkambi®, Elocta®, Mepolizumab Nucala®, Pitolisant, Efmoroctocog alfa, Etanercept, Oncaspar®, Wakix®, Numient®, Entresto®, Cobimetinib, Fentanilo, Praxbind®, Fenilbutirato de glycerol, Talimogene Laherparepvec, Levodopa/carbidopa, Blincyto®, Asparaginasa, Brivi®, Extracto de corteza de abedul, Ionsys®, Carfilzomib, Lumacaftor/Ivacaftor, Sacubitril/Valsartan, Benepali®, Kyprolis®, Imlygic®, Brivaracetam, Elvitegravir/Cobicistat/Emtricitabina/Tenofovir Alafenamida, Spectrila®, Spectrila, Episalvan®

وصف الملف: text/html

URL الوصول: https://explore.openaire.eu/search/publication?articleId=od_______624::3a967e98b652bd7edfc5f9eee6f29510
http://scielo.isciii.es/scielo.php?script=sci_arttext&pid=S1887-85712016000100007&lng=en&tlng=en

المؤلفون: Aparicio Hernández,R., García Luque,A., Gonzalo Salado,M.ªH., Cabanes Mariscal,M.A.

المصدر: Sanidad Militar v.72 n.1 2016

مصطلحات موضوعية: Asparaginasa, Spectrila, Blinatumomab, Blincyto®, Brivaracetam, Brivi®, Carfilzomib, Kyprolis®, Cobimetinib, Cotellic®, Efmoroctocog alfa, Elocta®, Elvitegravir/Cobicistat/Emtricitabina/Tenofovir Alafenamida, Genvoya®, Etanercept, Benepali®, Extracto de corteza de abedul, Episalvan®, Fenilbutirato de glycerol, Ravicti®, Fentanilo, Ionsys®, Idarucizumab, Praxbind®, Levodopa/carbidopa, Numient®, Lumacaftor/Ivacaftor, Orkambi®, Mepolizumab Nucala®, Pegaspargasa

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الاتاحة: https://scielo.isciii.es/scielo.php?script=sci_arttext&pid=S1887-85712016000100007

المؤلفون: Gentzsch, Martina, Ren, Hong Y., Houck, Scott A., Quinney, Nancy L., Cholon, Deborah M., Sopha, Pattarawut, Chaudhry, Imron G., Das, Jhuma, Dokholyan, Nikolay V., Randell, Scott H., Cyr, Douglas M.

المصدر: American Journal of Physiology - Lung Cellular and Molecular Physiology, 311(3)

مصطلحات موضوعية: Aminophenols, Sequence Deletion, cystic fibrosis, Quinolones, Cystic Fibrosis Transmembrane Conductance Regulator, W1282X, Aminopyridines, Orkambi, Drug Evaluation, Preclinical, Benzodioxoles, VX-770, Cell Line, heterozygote, Humans, Mutation, Missense, Protein Folding, rare mutation, R117H, VX-809

Relation: https://cdr.lib.unc.edu/downloads/9k41zk13f?file=thumbnail; https://cdr.lib.unc.edu/downloads/9k41zk13f

الاتاحة: https://doi.org/10.17615/pgag-s631
https://cdr.lib.unc.edu/downloads/9k41zk13f?file=thumbnail
https://cdr.lib.unc.edu/downloads/9k41zk13f