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Journal Articles

Mim8 prophylaxis in adults and adolescents with hemophilia A: 52-week efficacy and safety outcomes from the phase 3 FRONTIER2 study

Steven Lentz, Aby Abraham, Cihan Ay, Anthony Chan, Víctor Jiménez-Yuste, Johannes Oldenburg, Maria Elisa Mancuso, Johnny Mahlangu, Tadashi Matsushita, Lize van Vulpen, Renchi Yang, Amalie Rhode Høgh Nielsen, Ilgiz Rakhmatullin, Pratima Chowdary

Blood (2025) 146 (Supplement 1): 537.

https://doi.org/10.1182/blood-2025-537

Published: 2025

Journal Articles

Pregnancy and delivery outcomes in women with Hemophilia A and B: A multicenter cohort study from the Italian association of haemophilia centers

Mariasanta Napolitano, Marta Mattana, Claudia Cammarata, Marzia Leotta, Silvia Linari, Maria Elisa Mancuso, Gianna Franca Rivolta, Cristina Santoro, Sergio Siragusa, Lelia Valdrè, Rita Carlotta Santoro

Blood (2025) 146 (Supplement 1): 3073.

https://doi.org/10.1182/blood-2025-3073

Published: 2025

Journal Articles

NXT007 prophylaxis in people with hemophilia A with or without FVIII inhibitors: A global Phase I/II multiple-ascending-dose study

Maria Elisa Mancuso, Davide Matino, Dan Hart, Francisco-Jose Lopez-Jaime, Christophe Schmitt, Amy Shapiro, Giuliana Ventriglia, Anna Kiialainen, Laura Young, Olivier Catalani, Mark Belletrutti, Tom Chu, Jerzy Windyga, Víctor Jiménez-Yuste, Michaela Lehle, Janice Staber

Blood (2025) 146 (Supplement 1): 302.

https://doi.org/10.1182/blood-2025-302

Published: 2025

Journal Articles

Pharmacodynamic biomarkers in people with hemophilia A receiving multiple ascending doses of NXT007

Anna Kiialainen, Christophe Schmitt, Davide Matino, Janice Staber, Maria Elisa Mancuso, Víctor Jiménez-Yuste, Jerzy Windyga, Amy Shapiro, Matthew Locke, Dan Hart

Blood (2025) 146 (Supplement 1): 4841.

https://doi.org/10.1182/blood-2025-4841

Published: 2025

Journal Articles

Shaping individualized care for women with hemophilia A

Maria Elisa Mancuso

Blood (2025) 146 (16): 1880–1881.

https://doi.org/10.1182/blood.2025030645

Published: 2025

Journal Articles

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

Steven W. Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Sonia Deb, Markus Niggli, Tiffany Chang, Michaela Lehle, Karin Fijnvandraat

Blood (2024) 143 (14): 1355–1364.

https://doi.org/10.1182/blood.2023021832

Published: 2024

Includes: Supplemental data

Journal Articles

Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study

Steven Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Tiffany Chang, Michaela Lehle, Markus Niggli, Karin Fijnvandraat

Blood (2023) 142 (Supplement 1): 505.

https://doi.org/10.1182/blood-2023-177963

Published: 2023

Journal Articles

Physical Activity, Bleedings and Quality of Life in Patients with Haemophilia a without Inhibitors - a Multicenter, Observational Italian Study with a Wearable Device

Maria Elisa Mancuso, Chiara Biasoli, Renato Marino, Andrea Buzzi, Daniele Preti, Sara Bendinelli, Rosaria Tempre, Elena Pompeo, Luigi Sannino, Giacomo Siri, Giancarlo Castaman

Blood (2023) 142 (Supplement 1): 663.

https://doi.org/10.1182/blood-2023-179089

Published: 2023

Journal Articles

Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7

Anna Kiialainen, Steven Pipe, Karin Fijnvandraat, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Johannes Oldenburg, Maria Elisa Mancuso, Christophe Dhalluin, Michaela Lehle, Peter Collins

Blood (2023) 142 (Supplement 1): 1238.

https://doi.org/10.1182/blood-2023-173015

Published: 2023

Journal Articles

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Blood (2023) 142 (Supplement 1): 5480.

https://doi.org/10.1182/blood-2023-181299

Published: 2023

Journal Articles

Eptacog Beta Efficacy in Treating Mild or Moderate Bleeds in Target Joints of Individuals with Hemophilia A or B and Inhibitors in PERSEPT 1

Blood (2022) 140 (Supplement 1): 5597–5599.

https://doi.org/10.1182/blood-2022-156110

Published: 2022

Journal Articles

Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study

Steven W. Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Anna Kiialainen, Tiffany Chang, Michaela Lehle, Karin Fijnvandraat

Blood (2022) 140 (Supplement 1): 457–459.

https://doi.org/10.1182/blood-2022-157264

Published: 2022

Journal Articles

Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies

Blood (2021) 137 (16): 2231–2242.

https://doi.org/10.1182/blood.2020009217

Published: 2021

Includes: Supplemental data

Journal Articles

Potency-Adjusted Analyses of a Head-to-Head Pharmacokinetic Study of Damoctocog Alfa Pegol (BAY 94-9027) and Efmoroctocog Alfa (rFVIIIFc) in Patients with Severe Hemophilia A

Alexander Solms, Anita Shah, Sara Wiegmann, Maurice Ahsman, Erik Berntorp, Andreas Tiede, MD, Alfonso Iorio, MDPhD, Maria Elisa Mancuso, Tihomir Zhivkov, Toshko Lissitchkov

Blood (2020) 136 (Supplement 1): 24–25.

https://doi.org/10.1182/blood-2020-136586

Published: 2020

Journal Articles

BAY 94-9027 Provides Safe and Effective Long-Term Prophylaxis in Pediatric Patients: Results from the PROTECT VIII Kids Extension Study

Sanjay Ahuja, MD, Kathelijn Fischer, Tina Biss, Monika Maas Enriquez, Maria-Elisa Mancuso, MacGregor Steele, MD FRCPC, Maria Wang, Gili Kenet

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

https://doi.org/10.1182/blood-2020-140303

Published: 2020

Journal Articles

Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4)

Blood (2020) 136 (Supplement 1): 3–5.

https://doi.org/10.1182/blood-2020-137438

Published: 2020

Journal Articles

A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors

Guy Young, Ri Liesner, Tiffany Chang, Robert Sidonio, Jr, Johannes Oldenburg, Victor Jiménez-Yuste, Johnny Mahlangu, Rebecca Kruse-Jarres, Michael Wang, Marianne Uguen, Michelle Y. Doral, Lilyan Y. Wright, Christophe Schmitt, Gallia G. Levy, Midori Shima, Maria Elisa Mancuso

Blood (2019) 134 (24): 2127–2138.

https://doi.org/10.1182/blood.2019001869

Published: 2019

Includes: Supplemental data

Journal Articles

Emicizumab Prophylaxis Provides Flexible and Effective Bleed Control in Children with Hemophilia Α with Inhibitors: Results from the HAVEN 2 Study

Guy Young, Ri Liesner, Robert F. Sidonio, Jr., Johannes Oldenburg, Victor Jimenez-Yuste, Johnny Mahlangu, Rebecca Kruse-Jarres, Michael Wang, Tiffany Chang, Marianne Uguen, Michelle Doral, Christophe Schmitt, Gallia G. Levy, Midori Shima, MD PhD, Maria Elisa Mancuso

Blood (2018) 132 (Supplement 1): 632.

https://doi.org/10.1182/blood-2018-99-118153

Published: 2018

Journal Articles

Prospective Study of the Immunological Mechanisms of Immune Tolerance Induction in Severe Haemophilia a Patients with Inhibitors: Preliminary Analysis of a Multi-Center Longitudinal Study

Davide Matino, MD MSc, Giancarlo Castaman, Giovanni Di Minno, Paola Giordano, MD, Maria Elisa Mancuso, Emanuela Marchesini, Flora Peyvandi, MD PhD, Elena Santagostino, Cristina Santoro, MD PhD, Michele Schiavulli, Antonella De Luca, PhD, Marco Gargaro, Paolo Puccetti, MD, Francesca Fallarino, PhD, Alfonso Iorio, MD PhD

Blood (2018) 132 (Supplement 1): 3781.

https://doi.org/10.1182/blood-2018-99-119568

Published: 2018

Journal Articles

Prediction of Anti-FVIII Inhibitor Persistence By Anti-FVIII IgG Subclasses in Patients with Severe Hemophilia — A in the Sippet Cohort Study

Flora Peyvandi, MD PhD, Paolo Bucciarelli, MD, Carla Valsecchi, BSc, Marco Boscarino, Roberta Palla, PhD, Elena Santagostino, Amal El-Beshlawy, Mohsen Saleh Elalfy, MD, Vijay M. Ramanan, MBBS,MDDM, Peyman Eshghi, MD, Suresh Hanagavadi, MD, Mehran Karimi, MD, Cecil Ross, MD, Mamta V Manglani, MD, Guy Young, Tulika Seth, MDMBBS,FAAP, Shashikant Apte, MD FRCPA, Dinesh M Nayak, MD, Maria Elisa Mancuso, Johnny Mahlangu, Bonanad Santiago, MD, Monica H Cerqueira, MD PhD, Nadia P Ewing, MD, Christoph Male, MD, Tarek Owaidah, MDFRCPA, Veronica Soto Arellano, MD, Suvankar Majumdar, MBCHB, Mindy Simpson, MD, Mathew Thomas, MD, Ezio Zanon, Marilyn J Manco-Johnson, MD, Monica Martinez, MD, Maria Gabriella Mazzucconi, MD, Daniela Neme, MD, Rogelio Alejandro Paredes-Aguilera, MD, Alessandra N L Prezotti, MD MSc, Klaus Schmitt, MD, Bulent Zulfikar, Prof, Pier Mannuccio Mannucci, MD, Frits Richard Rosendaal, MD

Blood (2018) 132 (Supplement 1): 384.

https://doi.org/10.1182/blood-2018-99-114599

Published: 2018

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Mim8 prophylaxis in adults and adolescents with hemophilia A: 52-week efficacy and safety outcomes from the phase 3 FRONTIER2 study

Pregnancy and delivery outcomes in women with Hemophilia A and B: A multicenter cohort study from the Italian association of haemophilia centers

NXT007 prophylaxis in people with hemophilia A with or without FVIII inhibitors: A global Phase I/II multiple-ascending-dose study

Pharmacodynamic biomarkers in people with hemophilia A receiving multiple ascending doses of NXT007

Shaping individualized care for women with hemophilia A

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study

Physical Activity, Bleedings and Quality of Life in Patients with Haemophilia a without Inhibitors - a Multicenter, Observational Italian Study with a Wearable Device

Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Eptacog Beta Efficacy in Treating Mild or Moderate Bleeds in Target Joints of Individuals with Hemophilia A or B and Inhibitors in PERSEPT 1

Emicizumab Prophylaxis for the Treatment of Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Interim Analysis of the HAVEN 7 Study

Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies

Potency-Adjusted Analyses of a Head-to-Head Pharmacokinetic Study of Damoctocog Alfa Pegol (BAY 94-9027) and Efmoroctocog Alfa (rFVIIIFc) in Patients with Severe Hemophilia A

BAY 94-9027 Provides Safe and Effective Long-Term Prophylaxis in Pediatric Patients: Results from the PROTECT VIII Kids Extension Study

Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4)

A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors

Emicizumab Prophylaxis Provides Flexible and Effective Bleed Control in Children with Hemophilia Α with Inhibitors: Results from the HAVEN 2 Study

Prospective Study of the Immunological Mechanisms of Immune Tolerance Induction in Severe Haemophilia a Patients with Inhibitors: Preliminary Analysis of a Multi-Center Longitudinal Study

Prediction of Anti-FVIII Inhibitor Persistence By Anti-FVIII IgG Subclasses in Patients with Severe Hemophilia — A in the Sippet Cohort Study

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