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Journal Articles

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Steven W. Pipe, MD, Michael Recht, MD PhD, Nigel S. Key, MD, Frank W.G. Leebeek, MD PhD, Giancarlo Castaman, MD, Susan U. Lattimore, R.N., Paul Van Der Valk, Kathelijne Peerlinck, MD PhD, Michiel Coppens, MD, Niamh O'Connell, MD PhD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Peter Kampmann, MD, Karina Meijer, MD PhD, Annette von Drygalski, MD Pharm D, Guy Young, MD, Cedric Hermans, MDMRCP,PhD, Jan Astermark, MD PhD, Robert Klamroth, MD PhD, Richard S. Lemons, MD, Nathan Visweshwar, MD, Shelley Crary, MD MS, Rashid Kazmi, MBBS, Emily Symington, Miguel A. Escobar, MD, Esteban Gomez, MD, Rebecca Kruse-Jarres, MD, Adam Kotowski, MD, Doris Quon, MD PhD, Michael Wang, MD, Allison P. Wheeler, MD, Eileen K Sawyer, PhD, Stephanie Verweij, BSc, Valerie Colletta, MSc, Naghmana Bajma, MD, Robert Gut, MD PhD, Wolfgang A. Miesbach, MD PhD

Blood (2020) 136 (Supplement_2): LBA-6.

https://doi.org/10.1182/blood-2020-143560

Published: 2020

Journal Articles

Safety and Longer-Term Efficacy of Concizumab Prophylaxis in Patients with Hemophilia a or b with Inhibitors: Results from the Extension Part of the Phase 2 explorer4 Trial

Amy D Shapiro, MD, Giancarlo Castaman, MD, Katarina Cepo, MD, Sidsel Marie Tønder, MSc, Tadashi Matsushita, MD PhD, Lone Hvitfeldt Poulsen, Guy Young, MD, Silva Zupancic-Šalek, MD, Victor Jimenez Yuste, MD PhD

Blood (2020) 136 (Supplement 1): 40–41.

https://doi.org/10.1182/blood-2020-139910

Published: 2020

Journal Articles

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

Frank W.G. Leebeek, MD PhD, Karina Meijer, MD PhD, Michiel Coppens, MD, Peter Kampmann, MD, Robert Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Wolfgang A. Miesbach, MD

Blood (2020) 136 (Supplement 1): 26.

https://doi.org/10.1182/blood-2020-139225

Published: 2020

Journal Articles

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial

Annette von Drygalski, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MD, Susan U. Lattimore, Frank W.G. Leebeek, MD PhD, Wolfgang A. Miesbach, MD, Michael Recht, MD PhD, Esteban Gomez, MD, Robert Gut, Steven W. Pipe, MD

Blood (2020) 136 (Supplement 1): 13.

https://doi.org/10.1182/blood-2020-139295

Published: 2020

Journal Articles

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Wolfgang Miesbach, MD PhD, Karina Meijer, Michiel Coppens, MD, Peter Kampmann, MD, Dr. Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 2059.

https://doi.org/10.1182/blood-2019-122535

Published: 2019

Journal Articles

Bone and Joint Health Markers in Persons with Hemophilia A (PwHA) Treated with Emicizumab in HAVEN 3

Anna Kiialainen, PhD, Markus Niggli, PhD, Christine L. Kempton, MD MSc, Giancarlo Castaman, MD, Tiffany Y. Chang, MD MAS, Ido Paz-Priel, MD, Joanne I Adamkewicz, PhD, Gallia G. Levy, MD PhD

Blood (2019) 134 (Supplement_1): 626.

https://doi.org/10.1182/blood-2019-123689

Published: 2019

Journal Articles

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Hemophilia a or B with Inhibitors: Results from explorer4, a Phase 2, Randomized, Open-Label, Controlled Trial

Amy Shapiro, Giancarlo Castaman, MD, Katarina Cepo, Lone Hvitfeldt Poulsen, Christian Hollensen, Tadashi Matsushita, Guy Young, MD, Silva Zupancic-Salek, Victor Jimenez-Yuste

Blood (2019) 134 (Supplement_1): 1139.

https://doi.org/10.1182/blood-2019-122809

Published: 2019

Journal Articles

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Steven Pipe, MD, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MBChB, FRCP, Susan U Lattimore, Frank Leebeek, MD PhD, Wolfgang Miesbach, MD PhD, Michael Recht, MD PhD, Esteban Gomez, MD, Alison Long, Robert Gut, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 3348.

https://doi.org/10.1182/blood-2019-128765

Published: 2019

Journal Articles

Clinical, Instrumental, Serological and Histological Findings Suggest That Hemophilia B May be Less Severe Than Hemophilia A

Giancarlo Castaman, MD, Daniela Melchiorre, MD, Silvia Linari, MD

Blood (2015) 126 (23): 4682.

https://doi.org/10.1182/blood.V126.23.4682.4682

Published: 2015

Journal Articles

Short and Long-Term Outcomes in Hemophilia Patients with Inhibitors Undergoing Orthopedic Prosthetic Surgery

Giancarlo Castaman, MD, Silvia Linari, MD, Christian Carulli, MD, Massimo Innocenti, MD

Blood (2015) 126 (23): 4704.

https://doi.org/10.1182/blood.V126.23.4704.4704

Published: 2015

Journal Articles

Safety and Effectiveness of Desmopressin for the Management of Delivery and Major Surgery in Patients with Mild-Moderate Von Willebrand Disease: Final Analysis of the Prodeswil Study

Augusto Bramante Federici, MD, Giancarlo Castaman, MD, Alfonso Iorio, MD PhD, Emily Oliovecchio, Prodeswil Investigators

Blood (2015) 126 (23): 759.

https://doi.org/10.1182/blood.V126.23.759.759

Published: 2015

Journal Articles

Intracellular Retention, Enhanced Clearance, and Defective FVIII Binding Are Common Features of Von Willebrand Factor D'-D3 Domain Mutations in Patients with Von Willebrand Disease Type 1 From the European Mcmdm-1VWD Study

Reinhard Schneppenheim, MD, PhD, Ulrich Budde, MD, Javier Batlle, MD, Giancarlo Castaman, MD, Jeroen C. J. Eikenboom, MD, PhD, Augusto B. Federici, MD, Jenny Goudemand, MD, PhD, Tobias Obser, Florian Oyen, Francesco Rodeghiero, MD, Sonja Schneppenheim, MD, Ian R. Peake, PhD, Anne C. Goodeve, PhD

Blood (2012) 120 (21): 99.

https://doi.org/10.1182/blood.V120.21.99.99

Published: 2012

Journal Articles

Clinical Efficacy and Safety Versus Biological Response of Desmopressin (DDAVP) In Inherited Von Willebrand Disease (VWD) Types 1 and 2: Initial Results From the International Study Group on DDAVP In VWD In a Cohort of 229 Patients

Augusto B. Federici, MD, Alfonso Iorio, MD, Giancarlo Castaman, MD

Blood (2010) 116 (21): 238.

https://doi.org/10.1182/blood.V116.21.238.238

Published: 2010

Journal Articles

Reduced Von Willebrand Factor Secretion Is Associated with Loss of Weibel-Palade Formation

Giancarlo Castaman, MD, Sofia Helene Giacomelli, Paula M. Jacobi, BS, Tobias Obser, Reinhard Schneppenheim, MD, PhD, Sandra L Haberichter, PhD

Blood (2010) 116 (21): 541.

https://doi.org/10.1182/blood.V116.21.541.541

Published: 2010

Journal Articles

A Two-Year Prospective Study on Bleeding Tendency In 105 Patients with Type 2 A and M Von Willebrand Disease

Giancarlo Castaman, MD, Augusto B. Federici, MD, Luciano Baronciani, Pier M. Mannucci, MD, Francesco Rodeghiero

Blood (2010) 116 (21): 543.

https://doi.org/10.1182/blood.V116.21.543.543

Published: 2010

Journal Articles

Pharmacokinetic Evaluation of a Fibrinogen Concentrate in Patients with Congenital Fibrinogen Deficiency.

Marilyn J. Manco-Johnson, MD, Giancarlo Castaman, MD, Sandra Fremann, MD, Sigurd Knaub, PhD, Gavino Piseddu, MD, Pier M. Mannucci, MD

Blood (2008) 112 (11): 2268.

https://doi.org/10.1182/blood.V112.11.2268.2268

Published: 2008

Journal Articles

Type 2B “Malmo/New York” Is Not So Rare among Italian Patients with von Willebrand Disease: The Role of P1266L/Q Mutations of the von Willebrand Factor Gene in Seven Cases.

Luciano Baronciani, PhD, Silvia La Marca, PhD, Giancarlo Castaman, MD, Margherita Punzo, PhD, Rosanna Garavaglia, MD, Valentina Rubini, PhD, Giovanna Cozzi, PhD, Maria T. Canciani, PhD, Augusto B. Federici, MD

Blood (2007) 110 (11): 712.

https://doi.org/10.1182/blood.V110.11.712.712

Published: 2007

Journal Articles

Incidence and Determinants of Bleeding in Different Types of von Willebrand Disease: Results of the First Prospective Multicenter Study on 814 Italian Patients.

Augusto B. Federici, MD, Paolo Bucciarelli, MD, Giancarlo Castaman, MD, Maria G. Mazzucconi, MD, Massimo Morfini, MD, Angiola Rocino, MD, Mario Schiavoni, MD, Francesco Rodeghiero, MD, Pier M. Mannucci, MD

Blood (2007) 110 (11): 713.

https://doi.org/10.1182/blood.V110.11.713.713

Published: 2007

Journal Articles

Thrombotic and Hemorrhagic Complications after Surgery in Patients with Essential Thrombocythemia and Polycythemia Vera.

Marco Ruggeri, MD, Francesco Rodeghiero, MD, Alberto Tosetto, MD, Giancarlo Castaman, MD, Francesca Scognamiglio, MD, Guido Finazzi, MD, Federica Delaini, Caterina Micò, MD, Alessandro M. Vannucchi, MD, Elisabetta Antonioli, MD, Valerio De Stefano, MD, Tommaso Zà, MD, Luigi Gugliotta, MD, Alessia Tieghi, MD, Maria Gabriella Mazzucconi, MD, Cristina Santoro, MD, Tiziano Barbui, the GIMEMA MPD Working Party

Blood (2006) 108 (11): 2693.

https://doi.org/10.1182/blood.V108.11.2693.2693

Published: 2006

Journal Articles

F8 Gene Mutations and ITT Response in the Italian Cohort of Haemophilia A Patients with Inhibitors.

Giuseppe Tagariello, MD, Roberta Salviato, Donata Belvini, Paolo Radossi, Ezio Zanon, MD, Giorgio Gandini, MD, Giancarlo Castaman, MD, Roberto Sartori, MD

Blood (2005) 106 (11): 3211.

https://doi.org/10.1182/blood.V106.11.3211.3211

Published: 2005

NARROW

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Safety and Longer-Term Efficacy of Concizumab Prophylaxis in Patients with Hemophilia a or b with Inhibitors: Results from the Extension Part of the Phase 2 explorer4 Trial

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Bone and Joint Health Markers in Persons with Hemophilia A (PwHA) Treated with Emicizumab in HAVEN 3

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Hemophilia a or B with Inhibitors: Results from explorer4, a Phase 2, Randomized, Open-Label, Controlled Trial

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Clinical, Instrumental, Serological and Histological Findings Suggest That Hemophilia B May be Less Severe Than Hemophilia A

Short and Long-Term Outcomes in Hemophilia Patients with Inhibitors Undergoing Orthopedic Prosthetic Surgery

Safety and Effectiveness of Desmopressin for the Management of Delivery and Major Surgery in Patients with Mild-Moderate Von Willebrand Disease: Final Analysis of the Prodeswil Study

Intracellular Retention, Enhanced Clearance, and Defective FVIII Binding Are Common Features of Von Willebrand Factor D'-D3 Domain Mutations in Patients with Von Willebrand Disease Type 1 From the European Mcmdm-1VWD Study

Clinical Efficacy and Safety Versus Biological Response of Desmopressin (DDAVP) In Inherited Von Willebrand Disease (VWD) Types 1 and 2: Initial Results From the International Study Group on DDAVP In VWD In a Cohort of 229 Patients

Reduced Von Willebrand Factor Secretion Is Associated with Loss of Weibel-Palade Formation

A Two-Year Prospective Study on Bleeding Tendency In 105 Patients with Type 2 A and M Von Willebrand Disease

Pharmacokinetic Evaluation of a Fibrinogen Concentrate in Patients with Congenital Fibrinogen Deficiency.

Type 2B “Malmo/New York” Is Not So Rare among Italian Patients with von Willebrand Disease: The Role of P1266L/Q Mutations of the von Willebrand Factor Gene in Seven Cases.

Incidence and Determinants of Bleeding in Different Types of von Willebrand Disease: Results of the First Prospective Multicenter Study on 814 Italian Patients.

Thrombotic and Hemorrhagic Complications after Surgery in Patients with Essential Thrombocythemia and Polycythemia Vera.

F8 Gene Mutations and ITT Response in the Italian Cohort of Haemophilia A Patients with Inhibitors.

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