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

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

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

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

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

المؤلفون: Michael Laffan, K. John Pasi

المصدر: Postgraduate Haematology

مصطلحات موضوعية: medicine.medical_specialty, business.industry, Genetic enhancement, Internal medicine, medicine, Von Willebrand disease, Desmopressin, business, Haemophilia, medicine.disease, Gastroenterology, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::11ec1b7940793624748bc2fa95ebde02Test
https://doi.org/10.1002/9781118853771.ch38Test

المؤلفون: Hua Yu, Michael Laffan, Liron Walsh, Savita Rangarajan, David Perry, Glenn F. Pierce, Christian Vettermann, Wing Yen Wong, K. John Pasi, Will Lester, Bella Madan

مصطلحات موضوعية: 0301 basic medicine, Serotype, Adult, Male, medicine.medical_specialty, Genetic enhancement, VARIANT, Genetic Vectors, Hemorrhage, Antibodies, Viral, Hemophilia A, Gastroenterology, THERAPY, Virus, PROPHYLAXIS, 03 medical and health sciences, Young Adult, Medicine, General & Internal, hemic and lymphatic diseases, Internal medicine, General & Internal Medicine, medicine, MANAGEMENT, Humans, RATES, Young adult, Infusions, Intravenous, 11 Medical and Health Sciences, Factor IX, Science & Technology, Factor VIII, biology, business.industry, General Medicine, FACTOR-IX, Genetic Therapy, Dependovirus, Virus Shedding, Clinical trial, MICE, 030104 developmental biology, Cohort, DNA, Viral, biology.protein, TRIAL, Antibody, business, Life Sciences & Biomedicine, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4024b00e18bbd0f716af1b6f423e6c1dTest
http://hdl.handle.net/10044/1/57163Test

المؤلفون: Margaret V. Ragni, Michael Desmond Creagh, Akin Akinc, Inga Hegemann, Toshko Lissitchkov, Akshay Vaishnaw, K. John Pasi, Tim Mant, Pushkal Garg, Liana Gercheva-Kyuchukova, Margarita Timofeeva, Savita Rangarajan, Rashid S. Kazmi, Pencho Georgiev, Chang-Heok Soh, Steve Austin, David H. Bevan, Benny Sørensen, Charles R. M. Hay, Pratima Chowdary, Vasily Mamonov

المصدر: The New England journal of medicine. 377(9)

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Injections, Subcutaneous, Antithrombin III, 030204 cardiovascular system & hematology, Placebo, Hemophilia A, Gastroenterology, Hemophilia B, Antithrombins, law.invention, 03 medical and health sciences, Subcutaneous injection, Young Adult, 0302 clinical medicine, Randomized controlled trial, law, Internal medicine, medicine, Humans, Single-Blind Method, Clotting factor, Emicizumab, Dose-Response Relationship, Drug, business.industry, Antithrombin, Thrombin, General Medicine, Middle Aged, Healthy Volunteers, Surgery, Clinical trial, Dose–response relationship, RNAi Therapeutics, 030220 oncology & carcinogenesis, business, medicine.drug

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b158e5da40bdace27505aab605a9b2d9Test
https://pubmed.ncbi.nlm.nih.gov/28691885Test

المؤلفون: Pallavi Jain, Toshko Lissitchkov, Savita Rangarajan, Inga Hegemann, Meredith C. Foster, Craig Benson, Margaret V. Ragni, Syed Rashid Saeed Kazmi, Kate Madigan, K. John Pasi, Qifeng Yu, Michael Desmond Creagh, Huy Van Nguyen, Ketaki Kadam, Charles R. M. Hay, Pratima Chowdary, Pencho Georgiev, Steve Austin, Shariq Ali

المصدر: Blood. 132:2477-2477

مصطلحات موضوعية: 0301 basic medicine, Pediatrics, medicine.medical_specialty, business.operation, business.industry, Standard treatment, Immunology, Antithrombin, Cell Biology, Hematology, medicine.disease, Octapharma, Biochemistry, Discontinuation, Clinical trial, Bleeding diathesis, 03 medical and health sciences, 030104 developmental biology, medicine, In patient, Dosing, business, health care economics and organizations, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::66b23701311216d56fdc7f888e080790Test
https://doi.org/10.1182/blood-2018-99-112998Test

المؤلفون: K. John Pasi, Charlotte Warren, Simi Hurst

المصدر: Blood. 132:3485-3485

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, business.operation, business.industry, Immunology, Cell Biology, Hematology, Disease, 030105 genetics & heredity, Haemophilia, medicine.disease, Octapharma, medicine.disease_cause, Biochemistry, Clinical trial, 03 medical and health sciences, Hemophilias, Continuing medical education, Family medicine, medicine, business, Adeno-associated virus, Factor IX, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::bceaf65a3892a2bf1dd5da10e94094e0Test
https://doi.org/10.1182/blood-2018-99-119116Test