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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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المساهمون: Hematology
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 15, Iss, Pp 170-178 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy-Methods & Clinical Development, 15, 170-178. Cell Pressمصطلحات موضوعية: 0301 basic medicine, Serotype, lcsh:QH426-470, Transgene, viruses, Gene transfer, Bioinformatics, Article, Virus, 03 medical and health sciences, 0302 clinical medicine, Immune system, Genetics, Medicine, Vector (molecular biology), lcsh:QH573-671, Molecular Biology, business.industry, lcsh:Cytology, Clinical trial, lcsh:Genetics, 030104 developmental biology, Capsid, 030220 oncology & carcinogenesis, Molecular Medicine, business
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2a471130480982f1f71a50e6fac98c62Test
http://www.sciencedirect.com/science/article/pii/S2329050119300956Test -
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المؤلفون: K. John Pasi, P. Batty
المصدر: Expert Review of Precision Medicine and Drug Development. 4:259-262
مصطلحات موضوعية: Pharmacology, congenital, hereditary, and neonatal diseases and abnormalities, medicine.medical_specialty, business.industry, Genetic enhancement, Haemophilia A, Haemophilia, medicine.disease, Gastroenterology, Coagulation, hemic and lymphatic diseases, Internal medicine, Drug Discovery, Genetics, medicine, Molecular Medicine, Haemophilia B, business, Factor IX, medicine.drug
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::0dcccfbfcfd11cb978b0ca784b79d9f0Test
https://doi.org/10.1080/23808993.2019.1632704Test -
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المؤلفون: Tara M Robinson, Savita Rangarajan, K. John Pasi, Michael Laffan, Emily Symington, Will Lester, Bella Madan, Wing Yen Wong, Glenn F. Pierce, Xinqun Yang, Benjamin Kim, Nina Mitchell
المساهمون: Apollo - University of Cambridge Repository
المصدر: Haemophilia : the official journal of the World Federation of Hemophilia. 27(6)
مصطلحات موضوعية: FVIII, Male, congenital, hereditary, and neonatal diseases and abnormalities, medicine.medical_specialty, IMPACT, Genetic enhancement, Haemophilia A, haemophilia A, Hemorrhage, Haemophilia, Hemophilia A, Gastroenterology, PROPHYLAXIS, Hemostatics, Quality of life, QUALITY-OF-LIFE, hemic and lymphatic diseases, Internal medicine, medicine, Humans, Adverse effect, Genetics (clinical), Science & Technology, Factor VIII, business.industry, 1103 Clinical Sciences, ADULTS, Hematology, General Medicine, Genetic Therapy, medicine.disease, Confidence interval, Cardiovascular System & Hematology, Tolerability, Child, Preschool, haemostasis, Cohort, Quality of Life, business, Life Sciences & Biomedicine
وصف الملف: text/xml; application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0c531853ba9fa59e5941dee55d492e2dTest
https://pubmed.ncbi.nlm.nih.gov/34378280Test -
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المؤلفون: John Pasi, Desmond Creagh, D. J. Perry, Ruth Pink, Gregory M. Hayes, Amanda Clark, Sylvia Fong, Sophia Stanford, Gillian C. Lowe, Savita Rangarajan, Kandiah Chandrakumaran, Nicola Curry
المصدر: Research and Practice in Thrombosis and Haemostasis
مصطلحات موضوعية: Original Articles: Haemostasis, Serotype, seroprevalence, biology, business.industry, Genetic enhancement, adeno‐associated viral vectors, Hematology, Virus, Viral vector, Cohort, Immunology, biology.protein, Seroprevalence, Medicine, Original Article, hemophilia A, Vector (molecular biology), Antibody, business
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2476659e13f941e87501a741ca0983e5Test
https://doi.org/10.1002/rth2.12177Test -
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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