نتائج البحث

1

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

المساهمون: Vascular Medicine, ACS - Pulmonary hypertension & thrombosis, ACS - Amsterdam Cardiovascular Sciences, Real World Studies in PharmacoEpidemiology, -Genetics, -Economics and -Therapy (PEGET), Hematology

المصدر: New England journal of medicine, 388(8), 706-718. Massachussetts Medical Society
New England Journal of Medicine, 388(8), 706-718. MASSACHUSETTS MEDICAL SOC
New England Journal of Medicine, 388(8), 706-718. Massachussetts Medical Society

مصطلحات موضوعية: Coagulation, Genetics, General Medicine, Genetics General, Childhood Diseases, Hematology/Oncology, Pediatrics

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::18ec00c53efc81770ed02e6372363e8bTest
https://hdl.handle.net/11370/bcd8e7fb-66c2-4b0f-b235-d2995c4d444fTest

2

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

المصدر: New England Journal of Medicine. 386:1013-1025

مصطلحات موضوعية: Adult, Male, GENEr8-1 Trial Group, Factor VIII, Genetic Vectors, Alanine Transaminase, Hemorrhage, Genetic Therapy, General Medicine, Dependovirus, Hemophilia A, Intention to Treat Analysis, HIV Seronegativity, General & Internal Medicine, Humans, Infusions, Intravenous, 11 Medical and Health Sciences

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3b7596a3e58a0a0931c39ea2ce694c13Test
https://doi.org/10.1056/nejmoa2113708Test

3

Humanistic burden of problem joints for children and adults with haemophilia

المؤلفون: Tom Burke, Idaira Rodriguez‐Santana, Pratima Chowdary, Randall Curtis, Kate Khair, Michael Laffan, Paul Mclaughlin, Declan Noone, Brian O'Mahony, John Pasi, Mark Skinner, Jamie O'Hara

المصدر: Haemophilia : the official journal of the World Federation of HemophiliaREFERENCES.

مصطلحات موضوعية: Hematology, General Medicine, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::35794d1084a176cc6c32bfbf26f9b34fTest
https://pubmed.ncbi.nlm.nih.gov/36574369Test

4

Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table

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

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c38e32ba755156877323ace2a466b82dTest
https://doi.org/10.1111/hae.13971Test

5

Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study

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

6

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

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

7

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

المؤلفون: Sylvia Fong, Bridget Yates, Choong-Ryoul Sihn, Aras N. Mattis, Nina Mitchell, Su Liu, Chris B. Russell, Benjamin Kim, Adebayo Lawal, Savita Rangarajan, Will Lester, Stuart Bunting, Glenn F. Pierce, K. John Pasi, Wing Yen Wong

المصدر: Nature medicine. 28(4)

مصطلحات موضوعية: Factor VIII, Genetic Vectors, Humans, General Medicine, Genetic Therapy, RNA, Messenger, Transgenes, Dependovirus, Hemophilia A, General Biochemistry, Genetics and Molecular Biology

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::50ec81cb125a763b18f0efb8ec326442Test
https://pubmed.ncbi.nlm.nih.gov/35411075Test

8

Recombinant factor VIII Fc for the treatment of haemophilia A

المؤلفون: Cedric Hermans, Maria Elisa Mancuso, K. John Pasi, Beatrice Nolan

المساهمون: UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire, UCL - (SLuc) Service d'hématologie

المصدر: European Journal of Haematology
European journal of haematology, Vol. 106, no. 6, p. 745-761 (2021)

مصطلحات موضوعية: medicine.medical_specialty, immune tolerance, Recombinant Fusion Proteins, Haemophilia A, Once weekly, factor VIII‐Fc fusion protein, haemophilia A, Review Article, Haemophilia, Hemophilia A, Recombinant factor viii, 03 medical and health sciences, Therapeutic approach, 0302 clinical medicine, hemic and lymphatic diseases, half‐life, medicine, Humans, Factor VIII-Fc fusion protein, Intensive care medicine, surgical haemostasis, rFVIIIFc, Clinical Trials as Topic, Factor VIII, business.industry, Prophylaxis, Immune tolerance, Hematology, General Medicine, medicine.disease, Half-life, Immunoglobulin Fc Fragments, Fc fusion, Patient need, 030220 oncology & carcinogenesis, recombinant fusion proteins, prophylaxis, business, Dosing Frequency, 030215 immunology

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1f5f34e0da36f347a33816459b9017d8Test
https://pubmed.ncbi.nlm.nih.gov/33650192Test

9

Long‐term safety and sustained efficacy for up to 5 years of treatment with recombinant factor IX Fc fusion protein in subjects with haemophilia B: Results from the B‐YOND extension study

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

10

Lupus Anticoagulant and Abnormal Coagulation Tests in Patients with Covid-19

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 Medicine

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3880c064a315713c72ba84f22c469d42Test
http://europepmc.org/articles/PMC7217555Test

تنقيح النتائج