المؤلفون: Astermark, Jan, Buckner, Tyler W., Frenzel, Laurent, Hatswell, Anthony J., You, Xiaojun, Liu, Hai, Goodman, Erin, Santos, Sandra, Hawes, Charles, Hinds, David, Klamroth, Robert

المصدر: Haemophilia. 29(4):1087-1094

مصطلحات موضوعية: AAV5-hFVIII-SQ, emicizumab, FVIII prophylaxis, haemophilia A, matching-adjusted indirect comparison (MAIC), valoctocogene roxaparvovec, Medicin och hälsovetenskap, Klinisk medicin, Hematologi, Medical and Health Sciences, Clinical Medicine, Hematology

URL الوصول: https://lup.lub.lu.se/record/3b15c370-0c21-4382-a917-2cca499d9a3c
http://dx.doi.org/10.1111/hae.14818

المؤلفون: Agarwal, Suresh, Hermans, Cédric, Miesbach, Wolfgang, Peyvandi, Flora, Sidonio, Robert, Osmond, Dane, Newman, Vanessa, Henshaw, Josh, Pipe, Steven

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

المصدر: Haemophilia, , p. [1-9] (2024)

مصطلحات موضوعية: algorithm, emicizumab, gene therapy, haemophilia A, valoctocogene roxaparvovec

Relation: boreal:289827; http://hdl.handle.net/2078.1/289827; info:pmid/38684460; urn:ISSN:1351-8216; urn:EISSN:1365-2516

الاتاحة: http://hdl.handle.net/2078.1/289827
https://doi.org/10.1111/hae.15025

المؤلفون: Romeo Gabriel Mihaila

المصدر: Biomedical Papers, Vol 167, Iss 1, Pp 1-8 (2023)

مصطلحات موضوعية: adeno-associated viral vector, emicizumab, hemophilia a, lentiviral vector, valoctocogene roxaparvovec, Medicine

وصف الملف: electronic resource

Relation: https://biomed.papers.upol.cz/artkey/bio-202301-0001_from-a-bispecific-monoclonal-antibody-to-gene-therapy-a-new-era-in-the-treatment-of-hemophilia-a.php; https://doaj.org/toc/1213-8118; https://doaj.org/toc/1804-7521

URL الوصول: https://doaj.org/article/350b903fa11c474387c8340b39181f7a

المؤلفون: Lening Zhang, Bridget Yates, Ryan Murphy, Su Liu, Lin Xie, Britta Handyside, Choong-Ryoul Sihn, Taren Bouwman, Nicole Galicia, Danielle Tan, Carlos Fonck, Jeremy Arens, Annie Clark, Weiming Zhang, Sundeep Chandra, Jaydeep Srimani, Jennifer Holcomb, Andrea Van Tuyl, Joshua Henshaw, Christian Vettermann, Silvia Siso, Cheng Su, Sherry Bullens, Stuart Bunting, Charles O’Neill, Sylvia Fong

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 519-531 (2022)

مصطلحات موضوعية: AAV5, valoctocogene roxaparvovec, AAV5-hFVIII-SQ, neonates, gene transfer, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050122001127; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/19b2dc22c74b4524a6290d6684a6c08b

المؤلفون: Su Liu, Lisa Razon, Olivia Ritchie, Choong-Ryoul Sihn, Britta Handyside, Geoffrey Berguig, Jill Woloszynek, Lening Zhang, Paul Batty, David Lillicrap, Vishal Agrawal, Christa Cortesio, Kahsay Gebretsadik, Hassibullah Akeefe, Peter Colosi, Benjamin Kim, Stuart Bunting, Sylvia Fong

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 61-71 (2022)

مصطلحات موضوعية: AAV vectors, AAV transduction, hepatocytes, in vitro model, valoctocogene roxaparvovec, AAV5-hFVIII-SQ, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050122000730; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/a529774ab5f74b23856f5e350828dc5c

المؤلفون: Sylvia Fong, Britta Handyside, Choong-Ryoul Sihn, Su Liu, Lening Zhang, Lin Xie, Ryan Murphy, Nicole Galicia, Bridget Yates, Wesley C. Minto, Catherine Vitelli, Danielle Harmon, Yuanbin Ru, Guoying Karen Yu, Claudia Escher, Jakob Vowinckel, Jill Woloszynek, Hassib Akeefe, Rajeev Mahimkar, Sherry Bullens, Stuart Bunting

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 620-630 (2020)

مصطلحات موضوعية: AAV5, endoplasmic reticulum, hemophilia A, gene therapy, valoctocogene roxaparvovec, molecular chaperone, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050120301546; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/be656a12fe294471a8b8b8ad7da7d018

المؤلفون: Lening Zhang, Britta Handyside, Ryan Murphy, Choong-Ryoul Sihn, Lin Xie, Catherine Vitelli, Danielle Harmon, Sílvia Sisó, Su Liu, Sherry Bullens, Stuart Bunting, Sylvia Fong

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 13-20 (2020)

مصطلحات موضوعية: corticosteroids, prednisolone, Valoctocogene Roxaparvovec, FVIII expression, AAV, AAV5-hFVIII-SQ, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050119301329; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/dec3ee2ac6174f0a9f90c08723a1108e

المؤلفون: Quon, Doris V, Wang, Jiaan-Der, Wang, Michael, Pepperell, Dominic, Park, Young-Shil, Kenet, Gili, Mahlangu, Johnny, Khoo, Teh-Liane, Robinson, Tara M, Chavele, Konstantia-Maria, Pipe, Steven W

المصدر: Ther Adv Hematol ; ISSN:2040-6207 ; Volume:15

مصطلحات موضوعية: bleeding risk, gene therapy, hemophilia A, invasive procedures, valoctocogene roxaparvovec

Relation: https://doi.org/10.1177/20406207241304645; https://pubmed.ncbi.nlm.nih.gov/39712873; https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11660064/

الاتاحة: https://doi.org/10.1177/20406207241304645
https://pubmed.ncbi.nlm.nih.gov/39712873
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11660064/

المؤلفون: La Mura, Vincenzo, Cardinale, Vincenzo, De Cristofaro, Raimondo, De Santis, Adriano, Di Minno, Giovanni, Fabris, Luca, Marra, Fabio, Morisco, Filomena, Peyvandi, Flora, Pompili, Maurizio, Santoro, Cristina, Zanon, Ezio, Castaman, Giancarlo

المساهمون: La Mura, Vincenzo, Cardinale, Vincenzo, De Cristofaro, Raimondo, De Santis, Adriano, Di Minno, Giovanni, Fabris, Luca, Marra, Fabio, Morisco, Filomena, Peyvandi, Flora, Pompili, Maurizio, Santoro, Cristina, Zanon, Ezio, Castaman, Giancarlo

مصطلحات موضوعية: Patients with hemophilia A on valoctocogene roxaparvovec (gene therapy) must be managed carefully to minimize their risk of hepatotoxicity

Relation: info:eu-repo/semantics/altIdentifier/pmid/39226466; journal:BLOOD ADVANCES; https://hdl.handle.net/11573/1718129

الاتاحة: https://hdl.handle.net/11573/1718129
https://doi.org/10.1182/bloodadvances.2024013750

المؤلفون: O'Mahony, Brian, Dunn, Amy L., Leavitt, Andrew D., Peyvandi, Flora, Ozelo, Margareth C., Mahlangu, Johnny, Peerlinck, Kathelijne, Wang, Jiaan-Der, Lowe, Gillian C., Tan, Chee Wee, Giermasz, Adam, Tran, Huyen, Khoo, Teh-Liane, Cockrell, Erin, Pepperell, Dominic, Chambost, Hervé, López Fernández, Maria Fernanda, Kazmi, Rashid, Majerus, Elaine, Skinner, Mark W., Klamroth, Robert, Quinn, Jennifer, Yu, Hua, Wong, Wing Yen, Robinson, Tara M., Pipe, Steven W.

المساهمون: B. O'Mahony, A.L. Dunn, A.D. Leavitt, F. Peyvandi, M.C. Ozelo, J. Mahlangu, K. Peerlinck, J. Wang, G.C. Lowe, C.W. Tan, A. Giermasz, H. Tran, T. Khoo, E. Cockrell, D. Pepperell, H. Chambost, M.F. López Fernández, R. Kazmi, E. Majeru, M.W. Skinner, R. Klamroth, J. Quinn, Y. Hua, W.Y. Wong, T.M. Robinson, S.W. Pipe

مصطلحات موضوعية: gene therapy, health-related quality of life, hemophilia A, patient-reported outcome, valoctocogene roxaparvovec, Settore MED/09 - Medicina Interna

Relation: info:eu-repo/semantics/altIdentifier/pmid/37678546; info:eu-repo/semantics/altIdentifier/wos/WOS:001125013700001; volume:21; issue:12; firstpage:3450; lastpage:3462; numberofpages:13; journal:JOURNAL OF THROMBOSIS AND HAEMOSTASIS; https://hdl.handle.net/2434/1077088; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85173160645

الاتاحة: https://hdl.handle.net/2434/1077088
https://doi.org/10.1016/j.jtha.2023.08.032

المؤلفون: Choong-Ryoul Sihn, Britta Handyside, Su Liu, Lening Zhang, Ryan Murphy, Bridget Yates, Lin Xie, Richard Torres, Chris B. Russell, Charles A. O'Neill, Erno Pungor, Stuart Bunting, Sylvia Fong

المصدر: Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss, Pp 142-153 (2022)

مصطلحات موضوعية: QH573-671, QH426-470, circular episomes, AAV genome processing, AAV vectors, AAV5-hFVIII-SQ, hepatocyte-directed AAV gene transfer, Genetics, Molecular Medicine, Original Article, Cytology, Molecular Biology, oversized AAV vectors, valoctocogene roxaparvovec

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::65bc0f0e3dbe80f9ab4fa52e0ceb5aad
http://europepmc.org/articles/PMC8749450

المؤلفون: Hassib Akeefe, Su Liu, Jakob Vowinckel, Sherry Bullens, Choong-Ryoul Sihn, Lening Zhang, Ryan Murphy, Catherine Vitelli, Jill Woloszynek, Lin Xie, Bridget Yates, Guoying Karen Yu, Stuart Bunting, Danielle Harmon, Nicole Galicia, Rajeev Mahimkar, Wesley C. Minto, Britta Handyside, Sylvia Fong, Yuanbin Ru, Claudia Escher

المصدر: Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 620-630 (2020)

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, lcsh:QH426-470, Transgene, Genetic enhancement, Article, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, Genetics, medicine, Secretion, hybrid liver-specific promoter, 100ATGB, Vector (molecular biology), lcsh:QH573-671, Molecular Biology, valoctocogene roxaparvovec, lcsh:Cytology, Chemistry, Endoplasmic reticulum, RNA, Promoter, molecular chaperone, gene therapy, AAV5-hFVIII-SQ, lcsh:Genetics, endoplasmic reticulum, 030104 developmental biology, Endocrinology, 030220 oncology & carcinogenesis, Unfolded protein response, Molecular Medicine, AAV5, hemophilia A, ER stress

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::200f5b10877a9870dabd2d2acb6f3093
http://europepmc.org/articles/PMC7397702

المؤلفون: Britta Handyside, Sílvia Sisó, Catherine Vitelli, Lening Zhang, Danielle Harmon, Stuart Bunting, Sherry Bullens, Lin Xie, Ryan Murphy, Choong-Ryoul Sihn, Su Liu, Sylvia Fong

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 13-20 (2020)
Molecular Therapy. Methods & Clinical Development

مصطلحات موضوعية: 0301 basic medicine, congenital, hereditary, and neonatal diseases and abnormalities, lcsh:QH426-470, ALT, viruses, Transgene, Genetic enhancement, Pharmacology, FVIII expression, Severe hemophilia A, Article, Virus, corticosteroids, Clinical study, 03 medical and health sciences, 0302 clinical medicine, hemic and lymphatic diseases, Genetics, medicine, Vector (molecular biology), lcsh:QH573-671, AST, Molecular Biology, business.industry, lcsh:Cytology, prednisolone, AAV, Valoctocogene Roxaparvovec, AAV5-hFVIII-SQ, lcsh:Genetics, 030104 developmental biology, 030220 oncology & carcinogenesis, Prednisolone, Molecular Medicine, hemophilia A, business, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::49ce20f0311a4aac62525fa2b1279ffd
http://www.sciencedirect.com/science/article/pii/S2329050119301329

المؤلفون: Granda-Lobato,P, Correa-Pérez,A, Silva-Cuevas,M, García-Luque,A

المصدر: Sanidad Militar v.78 n.3 2022

مصطلحات موضوعية: Asciminib (Scemblix®), Budesonida (Kinpeygo®), Capmatinib (Travecta®), Efgartigimod Alfa (Vyvgart®), Eladocagene Exuparvovec (Upstaza®), Eptacog Beta Activado (Cevenfacta®), Extracto de corteza de abedul (Fisulvez®), Lasmiditan (Rayvow®), Lenacapavir (Sunlenca®), Melfalán Flufenamida (Pepaxti®), Mosunetuzumab (Lunsumio®), Olipudasa Alfa (Xenpozyme®), Lonafarnib (Zokinvy®), Vacuna frente a Covid-19 (inactivada, con adyuvante, adsorbida) (Valneva®), Valoctocogene Roxaparvovec (Roctavian®)

وصف الملف: text/html

الاتاحة: https://scielo.isciii.es/scielo.php?script=sci_arttext&pid=S1887-85712022000300010