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Journal Articles

Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec

Sylvia von Mackensen, Jonathan M. Ducore, Lindsey A. George, Adam Giermasz, Catherine McGuinn, John E. J. Rasko, Ben J Samelson-Jones, Spencer K. Sullivan, Jerome M. Teitel, Amit Chhabra, Annie F. Fang, Amanda O'Brien, Frank Plonski, Jeremy Rupon, Lynne Smith, Ian Winburn

Blood (2023) 142 (Supplement 1): 3628.

https://doi.org/10.1182/blood-2023-179431

Published: 2023

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

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Ben J. Samelson-Jones, Spencer K. Sullivan, John E.J. Rasko, Adam Giermasz, Lindsey A. George, Jonathan M. Ducore, Jerome M. Teitel, Catherine E. McGuinn, Amanda O'Brien, Ian Winburn, Lynne M Smith, Amit Chhabra, Jeremy Rupon

Blood (2021) 138 (Supplement 1): 3975.

https://doi.org/10.1182/blood-2021-150541

Published: 2021

Journal Articles

Single 225μg/Kg Dose Treatment with Eptacog Beta (Factor VIIa, Recombinant) Results in Rapid Hemostasis in Joint Bleeds for Persons with Hemophilia Α or Β with Inhibitors: A PERSEPT1 Subset Analysis

Janna M. Journeycake, MD, Cedric Hermans, MDMRCP,PhD, Jonathan M. Ducore, MD MPH, Miguel A. Escobar, MD, Guy Young, MD, Michael Wang, MD, Doris Quon, MD PhD, W Allan Alexander, MD, Ian S Mitchell, PhD, Ahmad Al-Sabbagh, MD, Daniel Bonzo, PhD

Blood (2020) 136 (Supplement 1): 2–3.

https://doi.org/10.1182/blood-2020-136443

Published: 2020

Journal Articles

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Lindsey A. George, MD, Spencer K. Sullivan, MD, John E.J. Rasko, MBBS, PhD, Adam Giermasz, MD PhD, Benjamin J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD MPH, Jerome M. Teitel, MD FRCPC, Catherine E. McGuinn, MD, Alexa R. Runowski, MS, Fraser Wright, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD, Denis Rybin, PhD, John E. Murphy, PhD, Jeremy Rupon, MD

Blood (2019) 134 (Supplement_1): 3347.

https://doi.org/10.1182/blood-2019-124091

Published: 2019

Journal Articles

Improvement in Quality of Life Outcomes and Bleeding Rates among Patients Switching from On-demand FVIII to Prophylactic BAY 94-9027 in the PROTECT VIII Study

Jonathan M. Ducore, MD MPH, Shadan Lalezari, PhD, Elena Santagostino, MD PhD, Sanjay P Ahuja, MD, Monika Maas Enriquez, Claudia Tueckmantel, PhD, Mark Reding

Blood (2019) 134 (Supplement_1): 1129.

https://doi.org/10.1182/blood-2019-123947

Published: 2019

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

A Novel Approach to Immune Tolerance Induction in Hemophilia Α with Factor VIII Inhibitor

Courtney D. Thornburg, MD, Jonathan M. Ducore, MD

Blood (2018) 132 (Supplement 1): 5042.

https://doi.org/10.1182/blood-2018-99-119800

Published: 2018

Journal Articles

Decrease in Overall and Joint Bleeding Rates with Extended-Interval Dosing: > 4 Years of Bay 94-9027 Prophylaxis in the Protect VIII Extension

Mark T. Reding, MD, Shadan Lalezari, Dr, Ingrid Pabinger, MD, Monica Maas Enriquez, Dr, Jonathan M. Ducore, MD

Blood (2018) 132 (Supplement 1): 1206.

https://doi.org/10.1182/blood-2018-99-116272

Published: 2018

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Daniel J Hui, PhD BS, Yun Liu, PhD, Ruchi Patel, Yifeng Chen, MD, Lindsey A. George, MD, Spencer K. Sullivan, MD, Adam Giermasz, MD, John Rasko, MBBS, PhD, Ben J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD, Sue Dasen, MS, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD

Blood (2017) 130 (Supplement 1): 2056.

https://doi.org/10.1182/blood.V130.Suppl_1.2056.2056

Published: 2017

Journal Articles

Assessing Safe Discharge Criteria for Pediatric Oncology Patients Admitted for Febrile Neutropenia

Emily Huschart, MD, Jong Chung, MD, Jonathan M. Ducore, MD

Blood (2017) 130 (Supplement 1): 4699.

https://doi.org/10.1182/blood.V130.Suppl_1.4699.4699

Published: 2017

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Blood (2017) 130 (Supplement 1): 601.

https://doi.org/10.1182/blood.V130.Suppl_1.601.601

Published: 2017

Journal Articles

Inhibitor Development in Previously Untreated Patients with Severe Hemophilia a Treated with Nuwiq ® , a New Generation Recombinant FVIII of Human Origin

Blood (2016) 128 (22): 327.

https://doi.org/10.1182/blood.V128.22.327.327

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

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression

Blood (2016) 128 (22): 3.

https://doi.org/10.1182/blood.V128.22.3.3

Published: 2016

NARROW

Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec

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

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Single 225μg/Kg Dose Treatment with Eptacog Beta (Factor VIIa, Recombinant) Results in Rapid Hemostasis in Joint Bleeds for Persons with Hemophilia Α or Β with Inhibitors: A PERSEPT1 Subset Analysis

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Improvement in Quality of Life Outcomes and Bleeding Rates among Patients Switching from On-demand FVIII to Prophylactic BAY 94-9027 in the PROTECT VIII Study

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

A Novel Approach to Immune Tolerance Induction in Hemophilia Α with Factor VIII Inhibitor

Decrease in Overall and Joint Bleeding Rates with Extended-Interval Dosing: > 4 Years of Bay 94-9027 Prophylaxis in the Protect VIII Extension

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Assessing Safe Discharge Criteria for Pediatric Oncology Patients Admitted for Febrile Neutropenia

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Inhibitor Development in Previously Untreated Patients with Severe Hemophilia a Treated with Nuwiq ® , a New Generation Recombinant FVIII of Human Origin

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

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression

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ASH Publications

American Society of Hematology

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ASH Publications

American Society of Hematology

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