المؤلفون: Oldenburg J; Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany. johannes.oldenburg@ukbonn.de., Chambost H; Department of Pediatric Hematology Oncology, AP-HM, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN, Marseille, France., Liu H; BioMarin Pharmaceutical Inc., Novato, CA, USA., Hawes C; BioMarin UK Ltd, London, UK., You X; BioMarin Pharmaceutical Inc., Novato, CA, USA., Yang X; BioMarin Pharmaceutical Inc., Novato, CA, USA., Newman V; BioMarin UK Ltd, London, UK., Robinson TM; BioMarin Pharmaceutical Inc., Novato, CA, USA., Hatswell AJ; DeltaHat Limited, Nottingham, UK.; Department of Statistical Science, University College London, London, UK., Hinds D; BioMarin Pharmaceutical Inc., Novato, CA, USA., Santos S; BioMarin UK Ltd, London, UK., Ozelo M; Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, SP, Brazil.

المصدر: Advances in therapy [Adv Ther] 2024 Jun; Vol. 41 (6), pp. 2267-2281. Date of Electronic Publication: 2024 Apr 15.

نوع المنشور: Journal Article; Multicenter Study; Clinical Trial, Phase III; Comparative Study

بيانات الدورية: Publisher: Health Communications Inc Country of Publication: United States NLM ID: 8611864 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1865-8652 (Electronic) Linking ISSN: 0741238X NLM ISO Abbreviation: Adv Ther Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/drug therapy , Hemophilia A*/complications , Factor VIII*/therapeutic use, Humans ; Male ; Adult ; Prospective Studies ; Female ; Middle Aged ; Hemorrhage/prevention & control ; Genetic Therapy/methods ; Treatment Outcome ; Young Adult

المؤلفون: Milani M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Canepari C; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Assanelli S; Department of Health Sciences, University of Piemonte Orientale, Novara, Italy., Merlin S; Department of Health Sciences, University of Piemonte Orientale, Novara, Italy., Borroni E; Department of Health Sciences, University of Piemonte Orientale, Novara, Italy., Starinieri F; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Biffi M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Russo F; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Fabiano A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Zambroni D; IRCCS San Raffaele Scientific Institute, Milan, Italy., Annoni A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Naldini L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Vita-Salute San Raffaele University, Milan, Italy., Follenzi A; Department of Health Sciences, University of Piemonte Orientale, Novara, Italy., Cantore A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy. cantore.alessio@hsr.it.; Vita-Salute San Raffaele University, Milan, Italy. cantore.alessio@hsr.it.

المصدر: EMBO molecular medicine [EMBO Mol Med] 2024 Jun; Vol. 16 (6), pp. 1427-1450. Date of Electronic Publication: 2024 Apr 29.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: EMBO Press Country of Publication: Germany NLM ID: 101487380 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1757-4684 (Electronic) Linking ISSN: 17574676 NLM ISO Abbreviation: EMBO Mol Med Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/therapy , Hemophilia A*/genetics , Genetic Vectors*/genetics , Endothelial Cells*/metabolism , Lentivirus*/genetics , Genetic Therapy*/methods , Liver*/metabolism , Factor VIII*/genetics , Factor VIII*/metabolism, Animals ; Mice ; Humans ; Viral Envelope Proteins/genetics ; Viral Envelope Proteins/metabolism ; Transduction, Genetic/methods ; Hepatocytes/metabolism ; Hepatocytes/virology ; Membrane Glycoproteins/genetics ; Membrane Glycoproteins/metabolism

المؤلفون: Jourdy Y; Hospices Civils de Lyon, Groupe Hospitalier Est, Service d'hématologie biologique, Bron, France; Université Claude Bernard Lyon 1, UR4609 Hémostase et thrombose, Lyon, France. Electronic address: yohann.jourdy@chu-lyon.fr., Chatron N; Hospices Civils de Lyon, Groupe Hospitalier Est, Service de génétique, Bron, France; Université Claude Bernard Lyon 1 - CNRS UMR 5261 -INSERM U1315, Institute NeuroMyoGène, Laboratoire Physiopathologie et Génétique du Neurone et du Muscle, Lyon, France., Frétigny M; Hospices Civils de Lyon, Groupe Hospitalier Est, Service d'hématologie biologique, Bron, France., Zawadzki C; Pôle de Biologie Pathologie Génétique, Institut d'Hématologie - Transfusion, CHU Lille, Lille, France., Lienhart A; Hospices Civils de Lyon, Lyon Hemophilia Center and Clinical Haemostasis Unit, Bron, France., Stieltjes N; Haemophilia Treatment Center, Cochin Hospital, AP-HP, Paris, France., Rohrlich PS; Service d'hématologie Clinique, CHU de Nice, Nice, France., Thauvin-Robinet C; Centre de Génétique, Centre de Référence, Déficiences Intellectuelles de Causes Rares, Hôpital d'Enfants, CHU Dijon Bourgogne, Dijon, France., Volot F; CRC-MHC Dijon, CHU Dijon, Dijon, France., Hamida YF; Laboratoire de recherche en hémostase, Université d'Alger 1, Alger, Algérie., Hariti G; Laboratoire de recherche en hémostase, Université d'Alger 1, Alger, Algérie., Leuci A; Université Claude Bernard Lyon 1, UR4609 Hémostase et thrombose, Lyon, France., Dargaud Y; Université Claude Bernard Lyon 1, UR4609 Hémostase et thrombose, Lyon, France; Hospices Civils de Lyon, Lyon Hemophilia Center and Clinical Haemostasis Unit, Bron, France., Sanlaville D; Hospices Civils de Lyon, Groupe Hospitalier Est, Service de génétique, Bron, France; Université Claude Bernard Lyon 1 - CNRS UMR 5261 -INSERM U1315, Institute NeuroMyoGène, Laboratoire Physiopathologie et Génétique du Neurone et du Muscle, Lyon, France., Vinciguerra C; Hospices Civils de Lyon, Groupe Hospitalier Est, Service d'hématologie biologique, Bron, France; Université Claude Bernard Lyon 1, UR4609 Hémostase et thrombose, Lyon, France.

المصدر: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Jun; Vol. 22 (6), pp. 1616-1626. Date of Electronic Publication: 2024 Mar 13.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/genetics , Hemophilia A*/diagnosis , Factor VIII*/genetics , Introns* , Chromosome Inversion* , Phenotype*, Humans ; Male ; Genetic Predisposition to Disease ; Severity of Illness Index ; Pedigree ; Chromosomes, Human, Pair 6/genetics ; DNA Mutational Analysis ; Chromosomes, Human, Pair 9/genetics ; Sequence Analysis, DNA ; Mutation ; Female

المؤلفون: Pezeshkpoor B; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany. Electronic address: Behnaz.pezeshkpoor@ukbonn.de., Berkemeier AC; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany., Herbst K; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany., Albert T; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany., Müller J; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany., Oldenburg J; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany; Center for Rare Diseases Bonn (ZSEB), University Clinic Bonn, Bonn, Germany. Electronic address: johannes.oldenburg@ukbonn.de.

المصدر: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Jun; Vol. 22 (6), pp. 1591-1604. Date of Electronic Publication: 2024 Mar 05.

نوع المنشور: Journal Article; Comparative Study; Evaluation Study

بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

مواضيع طبية MeSH: Factor VIII*/immunology , Hemophilia A*/immunology , Hemophilia A*/blood , Hemophilia A*/diagnosis , Immunoglobulin G*/blood , Immunoglobulin G*/immunology , Enzyme-Linked Immunosorbent Assay*/methods, Humans ; Immunoassay/methods ; Predictive Value of Tests ; Reproducibility of Results ; Male ; Protein Domains ; Antibodies, Neutralizing/blood ; Antibodies, Neutralizing/immunology ; Adolescent ; Microspheres

المؤلفون: Franchini M; Department of Transfusion Medicine and Hematology, Carlo Poma Hospital, Mantova, Italy., Focosi D; North-Western Tuscany Blood Bank, Pisa University Hospital, Pisa, Italy.

المصدر: Expert review of hematology [Expert Rev Hematol] 2024 Jun; Vol. 17 (6), pp. 233-240. Date of Electronic Publication: 2024 May 09.

نوع المنشور: Journal Article; Review

بيانات الدورية: Publisher: Taylor & Francis Country of Publication: England NLM ID: 101485942 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1747-4094 (Electronic) Linking ISSN: 17474094 NLM ISO Abbreviation: Expert Rev Hematol Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/drug therapy , Hemophilia A*/therapy , Hemophilia A*/immunology , Factor VIII*/therapeutic use , Factor VIII*/immunology , Immunosuppressive Agents*/therapeutic use, Humans ; Autoantibodies/immunology ; Rituximab/therapeutic use ; Disease Management

SCR Disease Name: Factor 8 deficiency, acquired

المؤلفون: Ceglédi A; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary.; Department of Public Health, Semmelweis University, Budapest, Hungary.; Doctoral College, Health Sciences Program, Semmelweis University, Budapest, Hungary., Bátai Á; Szent György Fejér County University Hospital, Székesfehérvár, Hungary., Dolgos J; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary., Fekete M; Department of Public Health, Semmelweis University, Budapest, Hungary., Gopcsa L; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary., Király V; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary., Lakatos G; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary., Nagy G; Department of Rheumatology and Clinical Immunology, Department of Internal Medicine and Oncology, Semmelweis University, Budapest, Hungary.; Heart and Vascular Center, Semmelweis University, Budapest, Hungary.; Hospital of the Hospitaller Order of Saint John of God, Budapest, Hungary., Szemlaky Z; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary., Várkonyi A; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary., Vilimi B; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary., Mikala G; Departments of Hematology and Stem Cell Transplantation, South Pest Central Hospital, National Institute of Hematology and Infectious Diseases, Budapest, Hungary.; School of PhD Studies, Semmelweis University, Budapest, Hungary., Bodó I; Department of Internal Medicine and Hematology, Semmelweis University, Budapest, Hungary.; Department of Hematology and Medical Oncology, Emory University, Atlanta, GA, United States.

المصدر: Pathology oncology research : POR [Pathol Oncol Res] 2024 May 23; Vol. 30, pp. 1611720. Date of Electronic Publication: 2024 May 23 (Print Publication: 2024).

نوع المنشور: Case Reports; Journal Article

بيانات الدورية: Publisher: Frontiers Media S. A Country of Publication: Switzerland NLM ID: 9706087 Publication Model: eCollection Cited Medium: Internet ISSN: 1532-2807 (Electronic) Linking ISSN: 12194956 NLM ISO Abbreviation: Pathol Oncol Res Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/drug therapy , Hemophilia A*/chemically induced , Adalimumab*/adverse effects , Adalimumab*/therapeutic use, Humans ; Male ; Aged ; Arthritis, Rheumatoid/drug therapy ; Factor VIIa/therapeutic use ; Antirheumatic Agents/therapeutic use ; Antirheumatic Agents/adverse effects ; Rituximab/therapeutic use ; Rituximab/adverse effects ; Retrospective Studies ; Recombinant Proteins/therapeutic use

SCR Disease Name: Factor 8 deficiency, acquired

المؤلفون: Kim AY; Research Institute of Pharmaceutical Science and Technology (RIPST), Ajou University, Suwon, South Korea; Department of Pediatrics, Gangnam Severance Hospital, Yonsei University College of Medicine, Seoul, South Korea., Baek HJ; Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, South Korea., Lee S; Research Institute of Pharmaceutical Science and Technology (RIPST), Ajou University, Suwon, South Korea; College of Pharmacy, Ajou University, Suwon, South Korea; Department of Biohealth Regulatory Science, Ajou University, Suwon, South Korea., Choo E; Research Institute of Pharmaceutical Science and Technology (RIPST), Ajou University, Suwon, South Korea., Park YS; Department of Pediatrics, Kyung Hee University College of Medicine, Kyung Hee University, Seoul, South Korea. Electronic address: pysped@khu.ac.kr., Lee H; Research Institute of Pharmaceutical Science and Technology (RIPST), Ajou University, Suwon, South Korea; College of Pharmacy, Ajou University, Suwon, South Korea; Department of Biohealth Regulatory Science, Ajou University, Suwon, South Korea. Electronic address: hankil@ajou.ac.kr.

المصدر: Thrombosis research [Thromb Res] 2024 May; Vol. 237, pp. 196-202. Date of Electronic Publication: 2024 Apr 13.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Pergamon Press Country of Publication: United States NLM ID: 0326377 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-2472 (Electronic) Linking ISSN: 00493848 NLM ISO Abbreviation: Thromb Res Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/economics , Hemophilia A*/immunology , Hemophilia A*/drug therapy , Immune Tolerance*, Humans ; Republic of Korea ; Male ; Child ; Adult ; Adolescent ; Child, Preschool ; Factor VIII/therapeutic use ; Factor VIII/immunology ; Factor VIII/economics ; Cost of Illness ; Young Adult ; Female ; Infant ; Health Care Costs

المؤلفون: Pierce GF; World Federation of Hemophilia, Montreal, Quebec, Canada. Electronic address: gpierce@wfh.org., Fong S; BioMarin Pharmaceutical Inc, Research and Early Development, Novato, California, USA., Long BR; BioMarin Pharmaceutical Inc, Research and Early Development, Novato, California, USA., Kaczmarek R; Department of Pediatrics, Indiana University School of Medicine, Wells Center for Pediatric Research, Indiana, USA; Laboratory of Glycobiology, Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Poland.

المصدر: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 May; Vol. 22 (5), pp. 1263-1289. Date of Electronic Publication: 2023 Dec 14.

نوع المنشور: Journal Article; Review; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural

بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE

مواضيع طبية MeSH: Dependovirus*/genetics , Hemophilia A*/therapy , Hemophilia A*/genetics , Genetic Therapy*/methods , Liver*/metabolism , Liver*/virology , Genetic Vectors*, Humans ; Animals ; Factor VIII/genetics ; Factor VIII/metabolism ; Gene Transfer Techniques

المؤلفون: Zangi AR; Student Research Committee, Tabriz University of Medical Sciences, Tabriz, Iran.; Department of Pharmaceutics, Faculty of Pharmacy, Tabriz University of Medical Sciences, Tabriz, 5166-15731, Iran., Amiri A; Department of Biotechnology, Faculty of Biological Sciences, Alzahra University, Tehran, Iran., Pazooki P; Cellular and Molecular Biology Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran., Soltanmohammadi F; Student Research Committee, Tabriz University of Medical Sciences, Tabriz, Iran.; Department of Pharmaceutics, Faculty of Pharmacy, Tabriz University of Medical Sciences, Tabriz, 5166-15731, Iran., Hamishehkar H; Drug Applied Research Center, Tabriz University of Medical Science, Tabriz, 5166-15731, Iran., Javadzadeh Y; Department of Pharmaceutics, Faculty of Pharmacy, Tabriz University of Medical Sciences, Tabriz, 5166-15731, Iran. javadzadehy@yahoo.com.

المصدر: Annals of hematology [Ann Hematol] 2024 May; Vol. 103 (5), pp. 1493-1511. Date of Electronic Publication: 2023 Nov 11.

نوع المنشور: Journal Article; Review

بيانات الدورية: Publisher: Springer Verlag Country of Publication: Germany NLM ID: 9107334 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1432-0584 (Electronic) Linking ISSN: 09395555 NLM ISO Abbreviation: Ann Hematol Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/therapy , Hemophilia A*/genetics, Humans ; Quality of Life ; Genetic Therapy/methods ; Factor VIII ; Blood Coagulation Factors ; Genetic Vectors

المؤلفون: Saiduzzaman M; Dr Md Saiduzzaman, Medical Officer, Department of Neurology, Mymensingh Medical College Hospital (MMCH), Mymensingh, Bangladesh; E-mail: munnam37@gmail.com., Hasan MT, Mia E

المصدر: Mymensingh medical journal : MMJ [Mymensingh Med J] 2024 Jul; Vol. 33 (3), pp. 929-931.

نوع المنشور: Case Reports; Journal Article

بيانات الدورية: Publisher: Mymensingh Medical College Country of Publication: Bangladesh NLM ID: 9601799 Publication Model: Print Cited Medium: Internet ISSN: 2408-8757 (Electronic) Linking ISSN: 10224742 NLM ISO Abbreviation: Mymensingh Med J Subsets: MEDLINE

مواضيع طبية MeSH: Hemophilia A*/complications , Hemophilia A*/diagnosis , Subarachnoid Hemorrhage*/etiology , Subarachnoid Hemorrhage*/complications, Humans ; Male ; Adolescent