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المؤلفون: Emily Symington, Glenn F. Pierce, Adam Giermasz, K. John Pasi, Stefan Tiefenbacher, Stephen J. Zoog, Mary Robinson, Savita Rangarajan, Steffen Rosen, Christian Vettermann, Donnie Mackenzie, Jaydeep K. Srimani, Benjamin Kim, Mei Huang, Terri Christianson
المصدر: Blood. 136:2524-2534
مصطلحات موضوعية: Male, congenital, hereditary, and neonatal diseases and abnormalities, Genetic enhancement, Transgene, Immunology, 030204 cardiovascular system & hematology, Pharmacology, Hemophilia A, Biochemistry, Virus, law.invention, 03 medical and health sciences, 0302 clinical medicine, Thrombin, Parvovirinae, law, hemic and lymphatic diseases, medicine, Humans, media_common.cataloged_instance, Transgenes, European union, media_common, Factor VIII, business.industry, Genetic Therapy, Cell Biology, Hematology, Dependovirus, Coagulation, Recombinant DNA, Specific activity, business, 030215 immunology, medicine.drug
وصف الملف: text
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b7b54fc4a67379b3e101a905a194db2aTest
https://doi.org/10.1182/blood.2020005683Test -
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المؤلفون: Glenn F. Pierce, Johnny Mahlangu, Thomas Sannié, David Lillicrap, Alok Srivastava, Dawn Rottellini, Donna Coffin, Thierry VandenDriessche, K. John Pasi, Radoslaw Kaczmarek, Alain Weill
المساهمون: Basic (bio-) Medical Sciences, Division of Gene Therapy & Regenerative Medicine
المصدر: Haemophilia. 26:443-449
مصطلحات موضوعية: medicine.medical_specialty, Consensus, media_common.quotation_subject, Genetic enhancement, haemophilia, 030204 cardiovascular system & hematology, Hemophilia A, Haemophilia, 03 medical and health sciences, 0302 clinical medicine, Multidisciplinary approach, Voting, medicine, Humans, Challenges, round table, Genetics (clinical), media_common, Medicine(all), business.industry, Health technology, Gene Therapy, Genetic Therapy, Hematology, General Medicine, global, Payment, medicine.disease, Clinical trial, Round table, Family medicine, business, 030215 immunology
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c38e32ba755156877323ace2a466b82dTest
https://doi.org/10.1111/hae.13971Test -
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المؤلفون: Bent Winding, Keiji Nogami, Guy Young, Chris Barnes, Huixing Yuan, Johannes Oldenburg, Elena Santagostino, Liane Khoo, Beatrice Nolan, Barbara A. Konkle, Joachim Fruebis, K. John Pasi, Ingrid Pabinger, Dan Rudin, Johnny Mahlangu
المصدر: Haemophilia
مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Adolescent, Recombinant Fusion Proteins, 030204 cardiovascular system & hematology, Haemophilia, Hemophilia A, Recombinant factor viii, bleed rate, 03 medical and health sciences, Young Adult, 0302 clinical medicine, Internal medicine, medicine, Clinical endpoint, Humans, Clinical Haemophilia, Child, Genetics (clinical), rFVIIIFc, Aged, extended half‐life, Factor VIII, business.industry, Hematology, General Medicine, Original Articles, Bleed, Middle Aged, medicine.disease, Confidence interval, Immunoglobulin Fc Fragments, Regimen, Fc fusion, Treatment Outcome, individualized prophylaxis, Child, Preschool, Severe haemophilia A, Original Article, Female, business, perioperative haemostasis, 030215 immunology
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bd4022ccca7aa7b264ac7e5d898662bfTest
http://europepmc.org/articles/PMC7384031Test -
4The impact of factor infusion frequency on health-related quality of life in people with haemophilia
المؤلفون: Xin Ying Lee, Gabriel Pedra, Sonia O’Hara, John Pasi, Pia Christoffersen, Kate Khair, Jamie O'Hara
المصدر: The Journal of Haemophilia Practice. 7:102-109
مصطلحات موضوعية: Health related quality of life, 03 medical and health sciences, medicine.medical_specialty, 0302 clinical medicine, business.industry, medicine, Pharmacy, 030204 cardiovascular system & hematology, business, Intensive care medicine, Haemophilia, medicine.disease, 030215 immunology
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::c42e32d1d3f9f4b3f0a07ab50f678970Test
https://doi.org/10.17225/jhp00158Test -
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المؤلفون: Roshni Kulkarni, Amy D. Shapiro, Bent Winding, Johan Szamosi, Margareth C. Ozelo, K. John Pasi, Stefan Lethagen, Christopher Barnowski
المصدر: Research and Practice in Thrombosis and Haemostasis
مصطلحات موضوعية: medicine.medical_specialty, 030204 cardiovascular system & hematology, 03 medical and health sciences, 0302 clinical medicine, Interquartile range, Internal medicine, medicine, Dosing interval, In patient, Factor IX, Original Articles: Haemostasis, factor IX, business.industry, Brief Report, clinical trial, Hematology, Bleed, Interim analysis, Clinical trial, 030220 oncology & carcinogenesis, Population study, hemophilia B, recombinant fusion proteins, prophylaxis, business, medicine.drug
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0b34eec4cf2d99c7830a720ab8d925d8Test
http://europepmc.org/articles/PMC6332734Test -
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المؤلفون: Liana Gercheva-Kyuchukova, Huy Van Nguyen, Tim Mant, Catherine Bagot, Baisong Mei, Qifeng Yu, Margaret V. Ragni, Craig Benson, Kate Madigan, Toshko Lissitchkov, K. John Pasi, Margarita Timofeeva, Pratima Chowdary, Pencho Georgiev, Vasily Mamonov
المصدر: Journal of Thrombosis and Haemostasis
مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Acetylgalactosamine, Erythema, 030204 cardiovascular system & hematology, Haemophilia, Hemophilia A, Gastroenterology, Hemophilia B, Antithrombins, fitusiran, 03 medical and health sciences, 0302 clinical medicine, Pharmacokinetics, Internal medicine, hemophilia, inhibitors, medicine, Humans, RNA, Small Interfering, Adverse effect, business.industry, HAEMOSTASIS, Antithrombin, Hematology, Original Articles, medicine.disease, antithrombin, Hemostasis, Pharmacodynamics, siRNA, Cohort, Quality of Life, Original Article, medicine.symptom, business, medicine.drug
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d8c4dd37dee07aac554599d82ced9e17Test
https://pubmed.ncbi.nlm.nih.gov/33587824Test -
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المؤلفون: Roshni Kulkarni, Johannes Oldenburg, Johnny Mahlangu, Hervé Chambost, Beatrice Nolan, Stephanie P’Ng, Carolyn M. Bennett, Margareth C. Ozelo, K. John Pasi, Bent Winding, Amy D. Shapiro, Huixing Yuan, Margaret V. Ragni, Joachim Fruebis, Tadashi Matsushita, Dan Rudin, Krista Fischer
المساهمون: Queen Mary's College [Chennai], University Medical Center [Utrecht], University of Pittsburgh (PITT), Pennsylvania Commonwealth System of Higher Education (PCSHE), Michigan State University System, University of Campinas [Campinas] (UNICAMP), University of Johannesburg (UJ), Indiana University - Purdue University Indianapolis (IUPUI), Indiana University System, Murdoch University, Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research (C2VN), Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE), Our Lady's Children's Hospital Crumlin (OLCHC), Emory University [Atlanta, GA], Nagoya City University [Nagoya, Japan], Stockholm University, Sanofi US, Institut für Genetik - Universität Bonn / Institute of Genetics - University of Bonn, Universidade Estadual de Campinas = University of Campinas (UNICAMP), University of Johannesburg [South Africa] (UJ), Lucas, Nelly
المصدر: Haemophilia
Haemophilia, Wiley, 2020, 26 (6), ⟨10.1111/hae.14036⟩
Haemophilia, 2020, 26 (6), ⟨10.1111/hae.14036⟩مصطلحات موضوعية: Adult, Male, [SDV.MHEP.HEM] Life Sciences [q-bio]/Human health and pathology/Hematology, medicine.medical_specialty, Time Factors, Adolescent, Recombinant Fusion Proteins, haemophilia B, 030204 cardiovascular system & hematology, Haemophilia, Hemophilia B, bleed rate, Young Adult, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, medicine, Clinical endpoint, Humans, Haemophilia B, extended half-life, Child, Genetics (clinical), Factor IX, factor IX, business.industry, [SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology, Hematology, General Medicine, Middle Aged, medicine.disease, Confidence interval, Immunoglobulin Fc Fragments, 3. Good health, Fc fusion, individualized prophylaxis, Child, Preschool, rFIXFc, Female, Long term safety, business, perioperative haemostasis, 030215 immunology, Recombinant factor IX, medicine.drug
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7cd16c5a39de08d060e95abeadfcd587Test
https://doi.org/10.1111/hae.14036Test -
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المؤلفون: K. John Pasi, Sean Platton, Daniel P. Hart, Suthesh Sivapalaratnam, Minal Dave, Vickie MacDonald, Kurtis Lee, Laura E. Green, Peter MacCallum, Louise Bowles, Nada Yartey
المصدر: The New England Journal of Medicine
New England Journal of Medicineمصطلحات موضوعية: medicine.medical_specialty, Coronavirus disease 2019 (COVID-19), 030204 cardiovascular system & hematology, Gastroenterology, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, Correspondence, Coagulation testing, Medicine, Humans, In patient, 030212 general & internal medicine, Pandemics, Blood coagulation test, Lupus anticoagulant, medicine.diagnostic_test, business.industry, SARS-CoV-2, COVID-19, General Medicine, medicine.disease, Antiphospholipid Syndrome, Pneumonia, Prolonged aptt, Lupus Coagulation Inhibitor, business, circulatory and respiratory physiology, Partial thromboplastin time
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3880c064a315713c72ba84f22c469d42Test
http://europepmc.org/articles/PMC7217555Test -
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المؤلفون: Tadbir K. Bariana, William J. Astle, Paquita Nurden, Jonathan Stephens, Sandra Le Quellec, Suthesh Sivapalaratnam, Andreas Greinacher, Jose A. Guerrero, Karyn Megy, Laxmikanth Kollipara, Hanna Shalev, Rachel Reed, Marie-Christine Alessi, Ron Kerr, Anthony D. Whetton, Matthew C Sims, Nihr BioResource, Man-Chiu Poon, Kathleen Freson, Samantha Farrow, Orna Steinberg-Shemer, Wendy N. Erber, Diane J. Nugent, Harriet McKinney, Cécile Lavenu-Bombled, Robert Campbell Tait, Mallika Sekhar, Rutendo Mapeta, Eva Leinoe, Anne M. Kelly, Louisa Mayer, Janine Collins, Mattia Frontini, Thierry M Leblanc, Elizabeth Chalmers, Albert Sickmann, Willem H. Ouwehand, Barbara Zieger, Taco W. Kuijpers, Antonio Rodriguez-Romera, Gian Marco Podda, Daniel P. Hart, Paolo Gresele, Daniel Greene, Keith Gomez, Wadie F. Bahou, Soo J. Park, Erica De Candia, Dave Lee, Luigi Grassi, Alan D. Michelson, Sara Morais, Denis Seyres, Kate Downes, John Pasi, Sri V V Deevi, John K. Wu, Loredana Bury, Frances Burden, Rachael Da Silva, Sofia Papadia, Marie-Françoise Hurtaud, Ernest Turro, Rémi Favier
المساهمون: Landsteiner Laboratory, Paediatric Infectious Diseases / Rheumatology / Immunology, AII - Inflammatory diseases, ARD - Amsterdam Reproduction and Development, University of Cambridge [UK] (CAM), CHU Trousseau [APHP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Leibniz-Institut für Analytische Wissenschaften - ISAS - e.V., University of Manchester [Manchester], Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research (C2VN), Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE), Stony Brook University [SUNY] (SBU), State University of New York (SUNY), University of Perugia, Royal Hospital for Sick Children [Edinburgh], Catholic University School of Medicine, Royal Free London NHS Foundation Trust, Universitätsmedizin Greifswald, Queen Mary University of London (QMUL), Hôpital Trousseau, Centre Hospitalier Régional Universitaire de Tours (CHRU Tours), Ninewells Hospital and Medical School [Dundee], Hospices Civils de Lyon (HCL), Rigshospitalet [Copenhagen], Copenhagen University Hospital, Harvard Medical School [Boston] (HMS), Centro Hospitalar Universitário do Porto, Children's Hospital of Orange County, University of California, Università degli Studi di Milano [Milano] (UNIMI), University of Calgary, Manchester Academic Health Science Centre (MAHSC), Ben-Gurion University of the Negev (BGU), Tel Aviv University [Tel Aviv], Glasgow Royal Infirmary, University of British Columbia (UBC), University of Freiburg [Freiburg], VU University Medical Center [Amsterdam], University of Aberdeen, Catholic University of Leuven - Katholieke Universiteit Leuven (KU Leuven), The University of Western Australia (UWA), Hôpital Xavier Arnozan, Università degli Studi di Perugia = University of Perugia (UNIPG), University of California (UC), Università degli Studi di Milano = University of Milan (UNIMI), Tel Aviv University (TAU), Prémilleux, Annick
المصدر: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Agência para a Sociedade do Conhecimento (UMIC)-FCT-Sociedade da Informação
instacron:RCAAP
Blood, 136(17), 1956-1967. American Society of Hematology
Blood
Blood, American Society of Hematology, 2020, 136 (17), pp.1956-1967. ⟨10.1182/blood.2019004776⟩
Blood, 2020, 136 (17), pp.1956-1967. ⟨10.1182/blood.2019004776⟩مصطلحات موضوعية: Biopsy, [SDV]Life Sciences [q-bio], 030204 cardiovascular system & hematology, medicine.disease_cause, GUIDELINES, NBEAL2, Biochemistry, Cohort Studies, 0302 clinical medicine, Megakaryocyte, Gene Frequency, Medicine, 0303 health sciences, [SDV.MHEP] Life Sciences [q-bio]/Human health and pathology, ABNORMALITIES, Hematology, Blood Proteins, ASSOCIATION, 3. Good health, [SDV] Life Sciences [q-bio], medicine.anatomical_structure, Phenotype, BEACH-DOMAIN, Immune System Diseases, medicine.symptom, BLOOD Commentary, Platelet disease, NEUROBEACHIN-LIKE 2, PROTEINS, Immunology, education, Inflammation, Cytoplasmic Granules, Gray Platelet Syndrome, CLASSIFICATION, Gray platelet syndrome, Diagnosis, Differential, 03 medical and health sciences, Genetic Heterogeneity, Immune system, Humans, Genetic Association Studies, 030304 developmental biology, Autoimmune disease, business.industry, Autoantibody, Grey platelet syndrome, Cell Biology, Immune dysregulation, medicine.disease, Bleeding disease, PROTEOME, Settore MED/15 - MALATTIE DEL SANGUE, Case-Control Studies, Immune System, Mutation, Bone marrow, business, PATHOGENICITY, [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology
وصف الملف: application/pdf; Print
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e84218b5ae93cc596770a017817d7a1dTest
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المؤلفون: Chris B. Russell, Nina Mitchell, K. John Pasi, Savita Rangarajan, Wing Yen Wong, Michael Laffan, Will Lester, Bella Madan, Glenn F. Pierce, Emily Symington, Mingjin Li
مصطلحات موضوعية: Oncology, Adult, Male, LIVER TRANSDUCTION, FVIII, medicine.medical_specialty, STRESS, VIRUS VECTOR GENOMES, viruses, Genetic enhancement, Genetic Vectors, MEDLINE, Hemorrhage, 030204 cardiovascular system & hematology, Hemophilia A, Virus, PREEXISTING IMMUNITY, 03 medical and health sciences, Dependovirus, Young Adult, 0302 clinical medicine, Medicine, General & Internal, Internal medicine, General & Internal Medicine, medicine, Humans, 030212 general & internal medicine, Transgenes, Young adult, Infusions, Intravenous, 11 Medical and Health Sciences, Factor VIII, Science & Technology, business.industry, Coagulants, Follow up studies, General Medicine, Genetic Therapy, FACTOR-IX, ASSOCIATION, Middle Aged, Clinical trial, MICE, business, Life Sciences & Biomedicine, Biomarkers, Follow-Up Studies
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a2fb2f488cde0f386819c674b197bcaaTest
http://hdl.handle.net/10044/1/75763Test