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Journal Articles

Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial

Blood (2024) 143 (22): 2256–2269.

https://doi.org/10.1182/blood.2023021864

Published: 2024

Includes: Supplemental data

Journal Articles

Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age

Blood (2021) 138 (Supplement 1): 3195.

https://doi.org/10.1182/blood-2021-147434

Published: 2021

Journal Articles

Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study

Claude Négrier, Johnny Mahlangu, Michaela Lehle, Pratima Chowdary, Olivier Catalani, Víctor Jiménez-Yuste, Benjamin M. Beckermann, Christophe Schmitt, Cedric Hermans, Giuliana Ventriglia, Jerzy Windyga, Anna Kiialainen, Roseline d'Oiron, Paul Moorehead, Vanda Teodoro, Amy D. Shapiro, Johannes Oldenburg

Blood (2021) 138 (Supplement 1): 343.

https://doi.org/10.1182/blood-2021-146009

Published: 2021

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

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

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Claude Négrier, Margaret V. Ragni, MDMPH, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Steven W. Pipe, MD, Gili Kenet, Savita Rangarajan, MBBS,MDFRCP,FRCPath, Salim Kichou, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 36–37.

https://doi.org/10.1182/blood-2020-136536

Published: 2020

Journal Articles

Real-World Clinical Management of Patients with Congenital Hemophilia and Inhibitors: Interim Analysis of the FEIBA Global Outcome Study (FEIBA GO)

Cedric Hermans, MDMRCP,PhD, Claude Négrier, Pål A Holme, Carmen Escuriola, Ana R Cid, Nataliya Kemenyash, MSc, Jaco Botha, Viridiana Cano-Garcia, Jerzy Windyga

Blood (2020) 136 (Supplement 1): 23–24.

https://doi.org/10.1182/blood-2020-139298

Published: 2020

Journal Articles

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Steven W. Pipe, MD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Toshko Lissitchkov, Margaret V. Ragni, MDMPH, Claude Négrier, Qifeng Yu, Stacey Poloskey, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-136501

Published: 2020

Journal Articles

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Steven Pipe, MD, Margaret V. Ragni, MD MPH, Claude Négrier, MD PhD, Qifeng Yu, PhD, Naghmana Bajwa, MD, John Caminis, MD, Baisong Mei, MD PhD, Shauna R. Andersson, MD PhD

Blood (2019) 134 (Supplement_1): 1138.

https://doi.org/10.1182/blood-2019-124108

Published: 2019

Journal Articles

Treatment of Haemophilia a (HA) Patients with Inhibitors: Immune Tolerance Induction with a Single Factor VIII/Von Willebrand Factor Concentrate in an Observational Immune Tolerance Induction Study (ObsITI)

C. Escuriola Ettingshausen, Erik Berntorp, MD PhD, Yesim Dargaud, MD PhD, Zeynep Gutowski, PhD, Claude Negrier, Anna Pavlova, MD PhD, Johannes Oldenburg

Blood (2018) 132 (Supplement 1): 2482.

https://doi.org/10.1182/blood-2018-99-114408

Published: 2018

Journal Articles

Characteristics of Bleed-Free Patients on Every-5-Day Dosing in the Protect VIII (BAY 94-9027) Study

Elena Santagostino, MD PhD, Bryce A. Kerlin, MD, Claude Negrier, Liu Tian, Dr, Jonathan M. Ducore, MD

Blood (2018) 132 (Supplement 1): 2486.

https://doi.org/10.1182/blood-2018-99-116031

Published: 2018

Journal Articles

The Highly Prevalent Deletions in F8 Intron 13 Found in French Mild Haemophilia a Patients Result of Both Founder Effect and Recurrent De Novo Events

Yohann Jourdy, PhD PharmD, Mathilde Fretigny, PharmD, Fanny Lassalle, PharmD, David Lillicrap, MD, Claude Negrier, Patrice Bouvagnet, MD, Christine Vinciguerra, PharmD

Blood (2018) 132 (Supplement 1): 2489.

https://doi.org/10.1182/blood-2018-99-111791

Published: 2018

Journal Articles

Efficacy of an Extended Half-Life GlycoPEGylated rFVIII (N8-GP): Pooled Analysis of ABR (Results from Two Clinical Trials)

Pratima Chowdary, MD, Lone Hvitfeldt Poulsen, Miguel A Escobar, MD, Susan Kearney, Meera B. Chitlur, MD, Robert Klamroth, Mudi Misgav, Claude Negrier, Wan Hui Ong Clausen, Frank Driessler, Andrea Landorph, Elena Santagostino

Blood (2018) 132 (Supplement 1): 1177.

https://doi.org/10.1182/blood-2018-99-110308

Published: 2018

Journal Articles

Genetic Fusion to Factor XIII B-Subunit Improves the Pharmacokinetic Properties of Factor IX

Sandra Le Quellec, MD, Nathalie Enjolras, PhD, Jean-Claude Bordet, Vladmir Jacqueline, Claude Negrier, MD, Yesim Dargaud, MD PhD

Blood (2017) 130 (Supplement 1): 2339.

https://doi.org/10.1182/blood.V130.Suppl_1.2339.2339

Published: 2017

Journal Articles

Use of Bypassing Agents Prior to and Post Bypassing Agent Dosing Guidance during Emicizumab Prophylaxis: Analyses from the HAVEN 1 Study

Michael U. Callaghan, Claude Negrier, Guy Young, Liane Khoo, Johnny Mahlangu, Jerzy Windyga, Johannes Oldenburg, Jin Xu, Olivier Catalani, Elina Asikanius, Joanne I. Adamkewicz, Midori Shima, MDPhD

Blood (2017) 130 (Supplement 1): 3668.

https://doi.org/10.1182/blood.V130.Suppl_1.3668.3668

Published: 2017

Journal Articles

Individually Tailored By-Passing Therapy for the Management of Breakthrough Bleeds in Hemophilia Patients with Inhibitors Receiving Prophylaxis with Emicizumab

Yesim Dargaud, MD PhD, Anne Lienhart, MD, Nathalie Enjolras, PhD, Claude Negrier, MD

Blood (2017) 130 (Supplement 1): 3675.

https://doi.org/10.1182/blood.V130.Suppl_1.3675.3675

Published: 2017

Journal Articles

Adeno-Associated Virus Mediated Gene Transfer with Hyperfunctional FIX-E410H Variant Improves Mean Factor IX Activity Levels in Hemophilia B Mice

Sandra Le Quellec, MD, Allison P Dane, PhD, Claude Negrier, Yesim Dargaud, MD PhD, Amit C Nathwani, MD PhD, Nathalie Enjolras, PhD

Blood (2017) 130 (Supplement 1): 4610.

https://doi.org/10.1182/blood.V130.Suppl_1.4610.4610

Published: 2017

Journal Articles

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

Claude Negrier, MD, Margaret V. Ragni, MD MPH, Pencho Georgiev, MD, Toshko Jelev Lissitchkov, MD PhD, Huy Van Nguyen, PharmD, Kate Madigan, K John Pasi

Blood (2017) 130 (Supplement 1): 2366.

https://doi.org/10.1182/blood.V130.Suppl_1.2366.2366

Published: 2017

Journal Articles

Thrombin Generation and Bleeding Phenotype during Personalized Prophylaxis with Recombinant Human FVIII in Previously Treated Patients with Severe Hemophilia A

Yesim G. Dargaud, MDPhD, Robert Klamroth, Luminita Rusen, MD, Jerzy Windyga, MD, Johann Bichler, PhD, Sigurd Knaub, Claude Negrier, MD

Blood (2016) 128 (22): 1410.

https://doi.org/10.1182/blood.V128.22.1410.1410

Published: 2016

Journal Articles

Feiba Global Outcome Study (FEIBA-GO): Long-Term Real World Data on Apcc (Feiba R ) in Patients with Inhibitors. First Demographic Data

Ana Rosa Cid, MD, Carmen Escuriola, MD, Cedric R. Hermans, MD PhD MRCP, Pal André Holme, PhD MD, Claude Negrier, MD, Savita Rangarajan, Angiola Rocino, Jerzy Windyga, MD, Alessandro Gringeri, MD MSc, Roberto Crea, DVM, MSc

Blood (2016) 128 (22): 5038.

https://doi.org/10.1182/blood.V128.22.5038.5038

Published: 2016

NARROW

Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial

Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age

Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study

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

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)

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Real-World Clinical Management of Patients with Congenital Hemophilia and Inhibitors: Interim Analysis of the FEIBA Global Outcome Study (FEIBA GO)

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Treatment of Haemophilia a (HA) Patients with Inhibitors: Immune Tolerance Induction with a Single Factor VIII/Von Willebrand Factor Concentrate in an Observational Immune Tolerance Induction Study (ObsITI)

Characteristics of Bleed-Free Patients on Every-5-Day Dosing in the Protect VIII (BAY 94-9027) Study

The Highly Prevalent Deletions in F8 Intron 13 Found in French Mild Haemophilia a Patients Result of Both Founder Effect and Recurrent De Novo Events

Efficacy of an Extended Half-Life GlycoPEGylated rFVIII (N8-GP): Pooled Analysis of ABR (Results from Two Clinical Trials)

Genetic Fusion to Factor XIII B-Subunit Improves the Pharmacokinetic Properties of Factor IX

Use of Bypassing Agents Prior to and Post Bypassing Agent Dosing Guidance during Emicizumab Prophylaxis: Analyses from the HAVEN 1 Study

Individually Tailored By-Passing Therapy for the Management of Breakthrough Bleeds in Hemophilia Patients with Inhibitors Receiving Prophylaxis with Emicizumab

Adeno-Associated Virus Mediated Gene Transfer with Hyperfunctional FIX-E410H Variant Improves Mean Factor IX Activity Levels in Hemophilia B Mice

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

Thrombin Generation and Bleeding Phenotype during Personalized Prophylaxis with Recombinant Human FVIII in Previously Treated Patients with Severe Hemophilia A

Feiba Global Outcome Study (FEIBA-GO): Long-Term Real World Data on Apcc (Feiba R ) in Patients with Inhibitors. First Demographic Data

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American Society of Hematology

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