المؤلفون: Klamroth, Robert, Hayes, Gregory, Andreeva, Tatiana, Gregg, Keith, Suzuki, Takashi, Mitha, Ismail Haroon, Hardesty, Brandon, Shima, Midori, Pollock, Toni, Slev, Patricia, Oldenburg, Johannes, Ozelo, Margareth C, Stieltjes, Natalie, Castet, Sabine-Marie, Mahlangu, Johnny, Peyvandi, Flora, Kazmi, Rashid, Schved, Jean-François, Leavitt, Andrew D, Callaghan, Michael, Pan-Petesch, Brigitte, Quon, Doris V, Andrews, Jayson, Trinh, Alex, Li, Mingjin, Wong, Yen

المصدر: Human Gene Therapy. 33(7-8)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Genetics, Prevention, Antibodies, Neutralizing, Antibodies, Viral, Dependovirus, Genetic Vectors, Hemophilia A, Humans, Prospective Studies, Seroepidemiologic Studies, Serogroup, gene therapy, hemophilia A, adeno-associated virus, antibody, seropositivity, Clinical Sciences, Biotechnology, Medical biotechnology

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/4fd5t2tvTest

المؤلفون: Noone Declan, Astermark Jan, O'Mahony Brian, Peyvandi Flora, Khair Kate, Pembroke Luke, Jenner Kathryn

المصدر: The Journal of Haemophilia Practice, Vol 9, Iss 1, Pp 156-166 (2023)

مصطلحات موضوعية: haemophilia, gene therapy, hub and spoke care model, patient-centred care, decision making, follow-up, Diseases of the blood and blood-forming organs, RC633-647.5

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/2055-3390Test

الوصول الحر: https://doaj.org/article/e2e005e6698d451397fe768b73cd3d4cTest

المؤلفون: Miesbach, Wolfgang, Boban, Ana, Chowdary, Pratima, Coppens, Michiel, Crato, Miguel, Jimenez‐Yuste, Victor, Klamroth, Robert, Makris, Mike, Mulders, Greta, Peyvandi, Flora

المصدر: Haemophilia; May2024, Vol. 30 Issue 3, p852-854, 3p

مصطلحات موضوعية: GENE therapy, DATABASES, TREATMENT effectiveness, HEMOPHILIA, BLOOD coagulation factor IX

المؤلفون: Miesbach, Wolfgang, Boban, Ana, Chowdary, Pratima, Coppens, Michiel, Crato, Miguel, Jimenez‐Yuste, Victor, Klamroth, Robert, Makris, Mike, Mulders, Greta, Peyvandi, Flora

المصدر: Haemophilia; May2024, Vol. 30 Issue 3, p855-857, 3p

مصطلحات موضوعية: GENE therapy, REGIONAL differences, HEMOPHILIA, HEALTH facilities

المؤلفون: Konkle, Barbara A.¹ (AUTHOR), Peyvandi, Flora² (AUTHOR), Coffin, Donna³ (AUTHOR) dcoffin@wfh.org, Naccache, Mayss³ (AUTHOR), Youttananukorn, Toong³ (AUTHOR), Pierce, Glenn F.³ (AUTHOR)

المصدر: Haemophilia. Jan2024, Vol. 30 Issue 1, p232-235. 4p.

مصطلحات موضوعية: *GENE therapy, *INTERNATIONAL organization, *HEMOPHILIA, *HEALTH facilities, *HEMOPHILIA treatment

الشركة/الكيان: EUROPEAN Medicines Agency

المؤلفون: Boban, Ana, Baghaei, Fariba, Karin, Fijnvandraat, Klamroth, Robert, Miesbach, Wolfgang, Stephensen, David, Kavanagh, Mary, Noone, Declan, Crato, Miguel, Peyvandi, Flora

المصدر: Haemophilia; Nov2023, Vol. 29 Issue 6, p1442-1449, 8p

مصطلحات موضوعية: HEALTH facilities, GENE therapy, HEMOPHILIA treatment, HEMOPHILIACS, ACCREDITATION

مصطلحات جغرافية: EUROPE

المؤلفون: Peyvandi, Flora, Lillicrap, David, Mahlangu, Johnny, McLintock, Claire, Pasi, K. John, Pipe, Steven W., Scales, Wendy, Srivastava, Alok, VandenDriessche, Thierry

المساهمون: F. Peyvandi, D. Lillicrap, J. Mahlangu, C. McLintock, K.J. Pasi, S.W. Pipe, W. Scale, A. Srivastava, T. VandenDriessche

مصطلحات موضوعية: gene therapy, hemophilia, knowledge gap, physician, respondent, survey, Settore MED/09 - Medicina Interna

العلاقة: info:eu-repo/semantics/altIdentifier/pmid/32548564; info:eu-repo/semantics/altIdentifier/wos/WOS:000521550000001; volume:2020; issue:4; firstpage:644; lastpage:651; numberofpages:8; journal:RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS; http://hdl.handle.net/2434/744886Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85096793640

الإتاحة: https://doi.org/10.1002/rth2.12326Test
http://hdl.handle.net/2434/744886Test

المؤلفون: Cutica, Ilaria, Mortarino, Mimosa, Garagiola, Isabella, Pravettoni, Gabriella, Peyvandi, Flora

المساهمون: I. Cutica, M. Mortarino, I. Garagiola, G. Pravettoni, F. Peyvandi

مصطلحات موضوعية: attitude, gene therapy, hemophilia, knowledge, questionnaire, Settore M-PSI/01 - Psicologia Generale

العلاقة: info:eu-repo/semantics/altIdentifier/pmid/38142845; firstpage:1; lastpage:8; numberofpages:8; journal:JOURNAL OF THROMBOSIS AND HAEMOSTASIS; https://hdl.handle.net/2434/1030169Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85182382314

الإتاحة: https://doi.org/10.1016/j.jtha.2023.12.018Test
https://hdl.handle.net/2434/1030169Test

المؤلفون: Castaman, Giancarlo, Di Minno, Giovanni, De Cristofaro, Raimondo, Peyvandi, Flora

المصدر: International Journal of Molecular Sciences; Sep2022, Vol. 23 Issue 18, pN.PAG-N.PAG, 18p

مصطلحات موضوعية: GENE therapy, BISPECIFIC antibodies, HEMOPHILIACS, BLOOD coagulation factor VIII, TRANSGENE expression

مصطلحات جغرافية: ITALY

المؤلفون: Konkle, Barbara, Pierce, Glen, Coffin, Donna, Naccache, Mayss, Clark, R. Cary, George, Lindsey, Iorio, Alfonso, O’mahony, Brian, Pipe, Steven, Skinner, Mark, Watson, Crystal, Peyvandi, Flora, Mahlangu, Johnny

مصطلحات موضوعية: gene therapy, core data, hemophilia, registry, safety, Internal Medicine and Specialties, Health Sciences

وصف الملف: application/pdf

العلاقة: Konkle, Barbara; Pierce, Glen; Coffin, Donna; Naccache, Mayss; Clark, R. Cary; George, Lindsey; Iorio, Alfonso; O’mahony, Brian; Pipe, Steven; Skinner, Mark; Watson, Crystal; Peyvandi, Flora; Mahlangu, Johnny (2020). "Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH." Journal of Thrombosis and Haemostasis 18(11): 3074-3077.; https://hdl.handle.net/2027.42/163440Test; Journal of Thrombosis and Haemostasis; Butterfield JSS, Hege KM, Herzog RW, Kaczmarek R. A molecular revolution in the treatment of hemophilia. Mol Ther. 2019; 28 ( 4 ): 997 - 1015.; Nathwani AC. Gene therapy for hemophilia. Hematology. 2019; 2019: 1 - 8.; Pierce GF, Iorio A. Past, present and future of haemophilia gene therapy: from vectors and transgenes to known and unknown outcomes. Haemophilia. 2018; 24 ( Suppl 6 ): 60 - 67.; Peyvandi F, Makris M, Collins P, et al. Minimal dataset for post- registration surveillance of new drugs in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2017; 15: 1878 - 1881.; Colella P, Ronzitti G, Mingozzi F. Emerging issues in AAV- mediated in vivo gene therapy. Mol Ther Methods Clin Dev. 2017; 8: 87 - 104.; George LA. Hemophilia gene therapy comes of age. Hematology. 2017; 2017: 587 - 594.; Konkle BA, Skinner M, Iorio A. Hemophilia trials in the twenty- first century: defining patient important outcomes. Res Pract Thromb Haemost. 2019; 3: 184 - 192.; Carmona G, Barnery L, Sewell J, et al. Correcting rare blood disorders using coagulation factors produced in vivo by shielded living therapeutics TM products. Blood. 2019; 134 ( Supplement 1 ): 2065.; Pasi KJ, Rangarajan S, Mitchell N, et al. Multiyear follow- up of AAV5- hFVIII- SQ gene therapy for hemophilia A. N Engl J Med. 2020; 382: 29 - 40.; Nguyen GN, Everett JK, Raymond H, et al. Long- term AAV- mediated factor VIII expression in nine hemophilia A dogs: a 10 year follow- up analysis on durability, safety and vector integration. Blood. 2019; 134 ( Suppl1 ): 611.; Chandler RJ, LaFave MC, Varshney GK, et al. Vector design influences hepatic genotoxicity after adeno- associated virus gene therapy. J Clin Invest. 2015; 125: 870 - 880.; Donsante A, Miller DG, Li Y, et al. AAV vector integration sites in mouse hepatocellular carcinoma. Science. 2007; 317: 477.; Gil- Farina I, Fronza R, Kaeppel C, et al. Recombinant AAV integration is not associated with hepatic genotoxicity in nonhuman primates and patients. Mol Ther. 2016; 24: 1100 - 1105.; Batty P, Lillicrap D. Advances and challenges for hemophilia gene therapy. Hum Mol Genet. 2019; 28: R95 - R101.; Peyvandi F, Garagiola I. Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia. Haemophilia. 2019; 25: 738 - 746.; Pharmacovigilance and epidemiology and regulatory and science management departments inspections HM, pharmacovigilance and committees and evaluation divisions. Report on Haemophilia Registries Workshop 8 June 2018. https://www.ema.europa.eu/en/documents/report/reportTest- haemophilia- registries- workshop_en.pdf: European Medicines Agency; 2018: 1 - 23.; Long term follow- up after administration of human gene therapy products guidance for industry. https://www.fda.gov/regulatoryTest- information/search- fda- guidance- documents/long- term- follow- after- administration- human- gene- therapy- products; Human gene therapy for hemophilia guidance for industry. https://www.fda.gov/regulatoryTest- information/search- fda- guidance- documents/human- gene- therapy- hemophilia; Ray WA, Stein CM. Reform of drug regulation - beyond an independent drug- safety board. N Engl J Med. 2006; 354: 194 - 201.

الإتاحة: https://doi.org/10.1111/jth.15023Test
https://hdl.handle.net/2027.42/163440Test