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Journal Articles

Real-World Use of Efanesoctocog Alfa in Adult Patients with Hemophilia a Including Those with a History of Factor VIII Inhibitor

Anya Parekh, Cody Angerman, Marvin Sanchez, NguyenHuong Doan, Anjlee Mahajan, Adam Giermasz

Blood (2024) 144 (Supplement 1): 2320.

https://doi.org/10.1182/blood-2024-203996

Published: 2024

Journal Articles

Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study

Andrew D. Leavitt, Barbara A. Konkle, Kimo C. Stine, Nathan Visweshwar, Thomas J. Harrington, Adam Giermasz, Steven Arkin, Annie Fang, Frank Plonski, Anne Yver, Florence Ganne, Delphine Agathon, Maria de los Angeles Resa, Li-Jung Tseng, Gregory Di Russo, Bettina M. Cockroft, Liching Cao, Jeremy Rupon

Blood (2024) 143 (9): 796–806.

https://doi.org/10.1182/blood.2022018971

Published: 2024

Includes: Supplemental data

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

HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Steven Pipe, Esteban Gomez, Cedric R. Hermans, Adam Giermasz, Peter Kampmann, Richard Lemons, Nicholas Galante, Sandra LeQuellec, Paul Monahan

Blood (2023) 142 (Supplement 1): 2256.

https://doi.org/10.1182/blood-2023-187059

Published: 2023

Journal Articles

Bleeding Outcomes in Participants with Factor VIII Activity <5 IU/DL Post-Gene Transfer in GENEr8-1

Johnny Mahlangu, Margareth Castro Ozelo, Chee Wee Tan, Adam Giermasz, Jiaan-Der Wang, Dane Osmond, Hua Yu, Tara M. Robinson

Blood (2023) 142 (Supplement 1): 2250.

https://doi.org/10.1182/blood-2023-180083

Published: 2023

Journal Articles

Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α

Thomas J. Harrington, Adam Giermasz, Nathan Visweshwar, Andrew D. Leavitt, Barbara A. Konkle, Jeremy Rupon, Gregory Dirusso, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie F. Fang, Steven Arkin

Blood (2023) 142 (Supplement 1): 1054.

https://doi.org/10.1182/blood-2023-179422

Published: 2023

Journal Articles

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α

Adam Giermasz, Nathan Visweshwar, Thomas J. Harrington, Andrew D Leavitt, Barbara Konkle, Jeremy Rupon, Gregory Di Russo, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie Fang, Steven Arkin

Blood (2022) 140 (Supplement 1): 7776–7777.

https://doi.org/10.1182/blood-2022-159421

Published: 2022

Journal Articles

Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

Amy L. Dunn, Yesim Dargaud, Yasmina L. Abajas, Manuel Carcao, Giancarlo Castaman, Adam Giermasz, Cédric Hermans, Magdalena D. Lewandowska, Johnny Mahlangu, Shannon L. Meeks, Wolfgang A. Miesbach, Michael Recht, Vanessa Salinas, Tammuella Chrisentery-Singleton, Hongying Wang, Ian S Mitchell, Guy Young

Blood (2022) 140 (Supplement 1): 8485–8488.

https://doi.org/10.1182/blood-2022-157343

Published: 2022

Journal Articles

A Preliminary Analysis of Athn 16: Safety of Coagulation Factor VIIa (recombinant)-Jncw for the Treatment of Bleeding Events in Patients with Congenital Hemophilia a or B with Inhibitors

Tammuella Chrisentery-Singleton, Lauren E. Amos, Daniel Bonzo, Miguel Escobar, Adam Giermasz, Emmanuelle Lagrue, Christine Knoll, Sonia Nasr, Michael Recht, Spencer Sullivan, Doris V. Quon, Mark T. Reding

Blood (2022) 140 (Supplement 1): 11360–11361.

https://doi.org/10.1182/blood-2022-163372

Published: 2022

Journal Articles

Athn 16: Safety of Coagulation Factor VIIa (recombinant)-Jncw for the Treatment of Bleeding Events in Patients with Congenital Hemophilia a or B with Inhibitors with or without Prophylactic Treatment

Tammuella Chrisentery-Singleton, W Allan Alexander, Ahmad Al-Sabbagh, Daniel Bonzo, Michael U. Callaghan, Miguel A. Escobar, Adam Giermasz, Nikki Hirsh, Janna Journeycake, Sonia Nasr, Doris V. Quon, Michael Recht

Blood (2021) 138 (Supplement 1): 3201.

https://doi.org/10.1182/blood-2021-146247

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

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Blood (2021) 138 (Supplement 1): 4916.

https://doi.org/10.1182/blood-2021-146021

Published: 2021

Journal Articles

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia a

Nathan Visweshwar, Thomas J. Harrington, Andrew D Leavitt, Barbara A. Konkle, Adam Giermasz, Kimo Stine, Jeremy Rupon, Gregory Di Russo, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie Fang, Steven Arkin

Blood (2021) 138 (Supplement 1): 564.

https://doi.org/10.1182/blood-2021-148651

Published: 2021

Journal Articles

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

Steffen Rosen, Stefan Tiefenbacher, Mary Robinson, Mei Huang, Jaydeep Srimani, Donnie Mackenzie, Terri Christianson, K. John Pasi, Savita Rangarajan, Emily Symington, Adam Giermasz, Glenn F. Pierce, Benjamin Kim, Stephen J. Zoog, Christian Vettermann

Blood (2020) 136 (22): 2524–2534.

https://doi.org/10.1182/blood.2020005683

Published: 2020

Includes: Supplemental data

Journal Articles

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults with Severe Hemophilia a

Andrew D. Leavitt, MD, Barbara A. Konkle, MD, Kimo Stine, MD, Nathan Visweshwar, MD, Thomas J. Harrington, MD, Adam Giermasz, MD PhD, Steven Arkin, MD, Annie Fang, MD PhD, Frank Plonski, RN, MA, Lynne Smith, MBA, Li-Jung Tseng, PhD MBA, Gregory Di Russo, MD, Bettina M Cockroft, MD, Jeremy Rupon, MD, Didier Rouy, MD PhD

Blood (2020) 136 (Supplement 1): 12.

https://doi.org/10.1182/blood-2020-137648

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

Updated Follow-up of the Alta Study, a Phase 1/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 Gene Therapy in Adult Patients with Severe Hemophilia A

Barbara A. Konkle, MD, Kimo Stine, MD, Nathan Visweshwar, MD, Thomas J. Harrington, MD, Andrew D. Leavitt, MD, Adam Giermasz, MD PhD, Steven Arkin, MD, Gregory Di Russo, MD, Ashley Snyder, PharmD, MPH, Adrian Woolfson, MD PhD, Didier Rouy, MD PhD

Blood (2019) 134 (Supplement_1): 2060.

https://doi.org/10.1182/blood-2019-122143

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

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

Preference for Emicizumab over Prior Factor Treatments: Results from the HAVEN 3 and HAVEN 4 Studies

Victor Jimenez-Yuste, Midori Shima, MD PhD, Ido Paz-Priel, Aric Parnes, Michaela Lehle, Adam Giermasz, Avrita Campinha-Bacote, Markus Niggli, Jerzy Windyga, Sammy Chebon, Peter Trask, Johnny Mahlangu, Steven Pipe, Johannes Oldenburg

Blood (2018) 132 (Supplement 1): 1187.

https://doi.org/10.1182/blood-2018-99-118132

Published: 2018

NARROW

Real-World Use of Efanesoctocog Alfa in Adult Patients with Hemophilia a Including Those with a History of Factor VIII Inhibitor

Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study

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

HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Bleeding Outcomes in Participants with Factor VIII Activity <5 IU/DL Post-Gene Transfer in GENEr8-1

Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α

Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

A Preliminary Analysis of Athn 16: Safety of Coagulation Factor VIIa (recombinant)-Jncw for the Treatment of Bleeding Events in Patients with Congenital Hemophilia a or B with Inhibitors

Athn 16: Safety of Coagulation Factor VIIa (recombinant)-Jncw for the Treatment of Bleeding Events in Patients with Congenital Hemophilia a or B with Inhibitors with or without Prophylactic Treatment

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

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia a

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults with Severe Hemophilia a

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

Updated Follow-up of the Alta Study, a Phase 1/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 Gene Therapy in Adult Patients with Severe Hemophilia A

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

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

Preference for Emicizumab over Prior Factor Treatments: Results from the HAVEN 3 and HAVEN 4 Studies

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American Society of Hematology

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American Society of Hematology

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