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  Gene therapy for hemophilia B

  Pier Mannuccio Mannucci

  12-12-2025

  https://doi.org/10.4081/btvb.2025.425

  641

  PDF: 78

  HTML: 92

• CO51 | Novel biomarkers of neurological damage in acute immune-mediated thrombotic thrombocytopenic purpura A. Truma^1,2, I. Mancini¹, F. Verde^2,3, J. A. Giannotta¹, A. Artoni¹, B. Ferrari¹, P. Agosti^1,2, M. Gagliardi¹, A. Ratti^3,4, N. Ticozzi^2,3, F. Peyvandi^1,2 | ¹Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan; ²Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan; ³Department of Neurology and Laboratory of Neuroscience, IRCCS Istituto Auxologico Italiano, Milan; ⁴Università degli Studi di Milano, Department of Medical Biotechnology and Translational Medicine, Milan, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.261

  533

  PDF: 1

• CO49 | The burden of cerebral small vessel disease in ITTP patients from acute phase to remission A. Truma^1,2, F. M. Lo Russo¹, G. Conte^1,2, I. Mancini¹, M. Abbattista¹, J. A. Giannotta¹, A. Artoni¹, B. Ferrari¹, P. Agosti^1,2, F. M. Triulzi^1,2, F. Peyvandi^1,2 | ¹Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan; ²Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.259

  570

  PDF: 1

• PO39 | Bleeding events in Von Willebrand disease type 1 yearly treatable with desmopressin A. Gringeri^1,2, P.M. Mannucci³, M. Gringeri⁴, G. Castaman⁵, F. Peyvandi^3,6 | ¹Fondazione Charta, Milan; ²Centro di Sanità Solidale, Lucca; ³Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan; ⁴Department of Medical Biotechnologies and Translational Medicine, Postgraduate School of Clinical Pharmacology and Toxicology, University of Milan; ⁵Centre for Bleeding Disorders and Coagulation, Careggi University Hospital, Firenze; ⁶Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Italy

  22-10-2025

  https://doi.org/10.4081/btvb.2025.302

  583

  PDF: 3
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  Bioequivalence of von Willebrand factor-containing concentrates: implications for the choice in patients with von Willebrand disease. A position paper from the Italian Association of Hemophilia Centers

  Ezio Zanon, Giancarlo Castaman, Augusto Bramante Federici, Maria Elisa Mancuso, Gianfranco Pasut, Alberto Tosetto, Angiola Rocino, Rita Carlotta Santoro

  26-01-2026

  https://doi.org/10.4081/btvb.2026.415

  1183

  PDF: 317

  HTML: 144
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  The landscape of rare coagulation factor deficiency management in Italy: a national hemophilia center survey

  Silvia Linari, Renato Marino, Marzia Leotta, Antonio Coppola, Patrizia Di Gregorio, Augusto Bramante Federici, Flora Peyvandi, Cristina Santoro, Ezio Zanon, Rita Carlotta Santoro

  05-11-2025

  https://doi.org/10.4081/btvb.2025.369

  1272

  PDF: 192

  HTML: 117

  Appendix: 108
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  Diagnostic and management practices for inherited fibrinogen disorders: a nationwide survey of Italian Hemophilia Treatment Centers

  Renato Marino, Silvia Linari, Marzia Leotta, Antonio Coppola, Chiara Biasoli, Maria Elisa Mancuso, Flora Peyvandi, Cristina Santoro, Ezio Zanon, Rita Carlotta Santoro

  02-12-2025

  https://doi.org/10.4081/btvb.2025.372

  820

  PDF: 102

  HTML: 107

  Supplementary: 64

  Appendix: 70
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  The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art

  Giovanni Di Minno, Gaia Spadarella, Ilenia Lorenza Calcaterra, Giancarlo Castaman, Paolo Simioni, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Matteo Di Minno

  04-09-2024

  https://doi.org/10.4081/btvb.2024.144

  1984

  PDF: 348

  SUPPLEMENTARY MATERIAL: 204