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Journal Articles

Safety and Outcome of Early Start of Emicizumab in Neonates and Infants with Severe Hemophilia Α, a Real-World Experience

Saad Ahmed, Mary Kavanagh, Imelda Kelly, Caitriona Ferry, Bertille Mullen, Bridin Brady, Ruth Hunter Nolan, Beatrice Nolan

Blood (2024) 144 (Supplement 1): 3976.

https://doi.org/10.1182/blood-2024-203316

Published: 2024

Journal Articles

Clinical Outcomes over 2 Years of Once-Weekly Efanesoctocog Alfa Treatment in Children with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study

Lynn Malec, Beatrice Nolan, Anthony K C Chan, Manuela Albisetti, Sheng-Chieh Chou, Bulent Zulfikar, Mindy Simpson, Liqi Feng, Helena Palmborg, Graham Neill, Lydia Abad-Franch, Sriya Gunawardena, Karin Fijnvandraat

Blood (2024) 144 (Supplement 1): 5495.

https://doi.org/10.1182/blood-2024-206915

Published: 2024

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

Fitusiran Prophylaxis Improves Health-Related Quality of Life in People with Hemophilia a or B, with or without Inhibitors: Results of ATLAS-PPX Study

Gili Kenet, Beatrice Nolan, Tadashi Matsushita, Guy Young, Ting Quan, Viridiana Cano, Christian Sussebach, Shauna Andersson, Baisong Mei, Marion Afonso, Kaan Kavakli

Blood (2022) 140 (Supplement 1): 7977–7978.

https://doi.org/10.1182/blood-2022-167169

Published: 2022

Journal Articles

The Emicizumab Switch: Real-World Data of 251 Pediatric Patients from the Pednet Registry

Konrad Van Der Zwet, Marloes de Kovel, Jayashree Motwani, Christel Van Geet, Beatrice Nolan, Heidi Glosli, Gili Kenet, Kathelijn Fischer

Blood (2022) 140 (Supplement 1): 8442–8443.

https://doi.org/10.1182/blood-2022-162181

Published: 2022

Journal Articles

First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results

Christoph Königs, Margareth C. Ozelo, Amy Dunn, Roshni Kulkarni, Beatrice Nolan, Simon A. Brown, Michele Schiavulli, Sriya Gunawardena, Sutirtha Mukhopadhyay, Deepthi Jayawardene, Bent Winding, Manuel Carcao

Blood (2022) 139 (26): 3699–3707.

https://doi.org/10.1182/blood.2021013563

Published: 2022

Includes: Supplemental data

Journal Articles

Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial

Beatrice Nolan, Michael Recht, Pablo Rendo, Aletta Falk, Meredith Foster, Sandra Casiano, Antoine Rauch, Amy D. Shapiro

Blood (2021) 138 (Supplement 1): 498.

https://doi.org/10.1182/blood-2021-148765

Published: 2021

Journal Articles

Similar Pattern of Increase in FIX Levels in Female Carriers and Males with Hemophilia B Leyden

Saad Z Ahmed, MBBS, Michelle O'Rourke, FACSLM, Vince Jenkins, PhD, Caitriona Keenan, PhD, Irene Ellen Regan, BSc, FRCPath, MBA, Beatrice Nolan, FRCPath

Blood (2020) 136 (Supplement 1): 19–20.

https://doi.org/10.1182/blood-2020-142091

Published: 2020

Journal Articles

99.3% of Inhibitors in Severe Hemophilia a Develop before Exposure Day 75. Time to Change Definition of Previously Treated Patients; Data from 1038 Patients with Severe Hemophilia a of the Pednet Registry

Blood (2018) 132 (Supplement 1): 2472.

https://doi.org/10.1182/blood-2018-99-114518

Published: 2018

Journal Articles

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

Margaret Ragni, Roshni Kulkarni, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Kathelijn Fischer, Johnny Mahlangu, Amy Shapiro, Beatrice Nolan, Johannes Oldenburg, Tadashi Matsushita, Annemieke Willemze, Huixing Yuan, Dan Rudin

Blood (2018) 132 (Supplement 1): 1214.

https://doi.org/10.1182/blood-2018-99-110100

Published: 2018

Journal Articles

ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A

Beatrice Nolan, Johnny Mahlangu, Guy Young, Barbara A Konkle, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Johannes Oldenburg, MD PhD, Keiji Nogami, MDPhD, Nikola Tripkovic, Huixing Yuan, Dan Rudin

Blood (2018) 132 (Supplement 1): 1192.

https://doi.org/10.1182/blood-2018-99-118586

Published: 2018

Journal Articles

Longitudinal Analysis of Long-Term Safety and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Children with Severe Hemophilia a

Guy Young, Raina Liesner, K John Pasi, Beatrice Nolan, Stefan Lethagen, Lynda M Cristiano, Elisa Tsao, Bent Winding, Johnny Mahlangu

Blood (2016) 128 (22): 1414.

https://doi.org/10.1182/blood.V128.22.1414.1414

Published: 2016

Journal Articles

Risk Factors for the Development of High-Titer Inhibitors in 260 Children with Severe Hemophilia a Born Between 1990 and 2009: The Remain Study

Blood (2016) 128 (22): 3774.

https://doi.org/10.1182/blood.V128.22.3774.3774

Published: 2016

Journal Articles

Long-Term Efficacy and Quality of Life with Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia a

Michael Wang, MD, K John Pasi, Ingrid Pabinger, Prof, Bryce A. Kerlin, Roshni Kulkarni, MD, Beatrice Nolan, Raina Liesner, Simon A Brown, Hideji Hanabusa, Elisa Tsao, Bent Winding, Stefan Lethagen, Nisha Jain

Blood (2016) 128 (22): 3791.

https://doi.org/10.1182/blood.V128.22.3791.3791

Published: 2016

Journal Articles

Long Term Safety and Efficacy of Recombinant Factor IX Fc Fusion Protein (rFIXFc) Prophylaxis in Children with Hemophilia B: Interim Results of the B-Yond Extension Study

Jonathan M. Ducore, Kathelijn Fischer, Roshni Kulkarni, MD, Beatrice Nolan, Carolyn M. Bennett, David J Perry, Huixing Yuan, Alejandra Ramirez-Santiago, Francesca Ferrante, Stefan Lethagen

Blood (2016) 128 (22): 4976.

https://doi.org/10.1182/blood.V128.22.4976.4976

Published: 2016

Journal Articles

Clinical Outcomes in Children with Hemophilia B Treated Long Term with rFIXFc: Interim Results of the B-YOND Extension Study

Roshni Kulkarni, Beatrice Nolan, Carolyn M. Bennett, Kathelijn Fischer, David J Perry, Christopher Barnes, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Baisong Mei

Blood (2015) 126 (23): 1093.

https://doi.org/10.1182/blood.V126.23.1093.1093

Published: 2015

Journal Articles

Long-Term Efficacy of rFVIIIFc Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia A

Bryce A. Kerlin, Roshni Kulkarni, Beatrice Nolan, Michael Wang, MD, K John Pasi, Raina Liesner, Simon Brown, Hideji Hanabusa, Ingrid Pabinger-Fasching, Elisa Tsao, Geoffrey Allen

Blood (2015) 126 (23): 3520.

https://doi.org/10.1182/blood.V126.23.3520.3520

Published: 2015

Journal Articles

Safety, Efficacy, and Pharmacokinetics of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously-Treated Children with Severe Hemophilia a (Kids-ALONG)

Guy Young, MD, Johnny N. Mahlangu, MBBCh, MMed, FCPath, Roshni Kulkarni, MD, Beatrice Nolan, MB, MRCPI, FRCPATH, Raina Liesner, MA MBBChir MDFRCP FRCPath MRCPCH, John Pasi, MBChB, PhD FRCP, FRCPath, Christopher Barnes, MB BS, Srividya Neelakantan, PhD, Giulia Gambino, MSc, Lynda M Cristiano, MD, Christopher Barnowski, MD, Glenn F Pierce, MD PhD, Geoffrey A. Allen, MD

Blood (2014) 124 (21): 1494.

https://doi.org/10.1182/blood.V124.21.1494.1494

Published: 2014

Journal Articles

Pharmacokinetics Of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Pediatric Subjects With Hemophilia A: An Interim Analysis Of The Kids A-LONG Study

Guy Young, MD, Johnny Mahlangu, MD, Beatrice Nolan, MB, MRCPI, FRCPATH, Simon Brown, MD, FRACP, FRCPA, MBBS, FRCP, FRCPath, Leonard A. Valentino, MD, FAAP, Raina Liesner, MA, MBBChir, MD, FRCP, FRCPath, MRCPCH, Yingwen Dong, PhD, Lei Diao, PhD, Haiyan Jiang, PhD, Karen Nugent, MSc, Glenn F. Pierce, MD, PhD, Geoffrey Allen, MD

Blood (2013) 122 (21): 3609.

https://doi.org/10.1182/blood.V122.21.3609.3609

Published: 2013

Journal Articles

Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study

Blood (2013) 121 (20): 4046–4055.

https://doi.org/10.1182/blood-2012-09-457036

Published: 2013

Includes: Supplemental data

NARROW

Safety and Outcome of Early Start of Emicizumab in Neonates and Infants with Severe Hemophilia Α, a Real-World Experience

Clinical Outcomes over 2 Years of Once-Weekly Efanesoctocog Alfa Treatment in Children with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study

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

Fitusiran Prophylaxis Improves Health-Related Quality of Life in People with Hemophilia a or B, with or without Inhibitors: Results of ATLAS-PPX Study

The Emicizumab Switch: Real-World Data of 251 Pediatric Patients from the Pednet Registry

First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results

Prophylaxis with rFIXFc Reduces the Frequency and Delays Time to First Spontaneous Bleed Event in Previously Untreated Patients with Hemophilia B: A Post Hoc Analysis of the PUPs B-LONG Trial

Similar Pattern of Increase in FIX Levels in Female Carriers and Males with Hemophilia B Leyden

99.3% of Inhibitors in Severe Hemophilia a Develop before Exposure Day 75. Time to Change Definition of Previously Treated Patients; Data from 1038 Patients with Severe Hemophilia a of the Pednet Registry

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A

Longitudinal Analysis of Long-Term Safety and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Children with Severe Hemophilia a

Risk Factors for the Development of High-Titer Inhibitors in 260 Children with Severe Hemophilia a Born Between 1990 and 2009: The Remain Study

Long-Term Efficacy and Quality of Life with Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia a

Long Term Safety and Efficacy of Recombinant Factor IX Fc Fusion Protein (rFIXFc) Prophylaxis in Children with Hemophilia B: Interim Results of the B-Yond Extension Study

Clinical Outcomes in Children with Hemophilia B Treated Long Term with rFIXFc: Interim Results of the B-YOND Extension Study

Long-Term Efficacy of rFVIIIFc Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia A

Safety, Efficacy, and Pharmacokinetics of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously-Treated Children with Severe Hemophilia a (Kids-ALONG)

Pharmacokinetics Of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Pediatric Subjects With Hemophilia A: An Interim Analysis Of The Kids A-LONG Study

Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study

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American Society of Hematology

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American Society of Hematology

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