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Journal Articles

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 ΑFFINE Gene Therapy Trial

Blood (2024) 144 (Supplement 1): 1053.

https://doi.org/10.1182/blood-2024-208486

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

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

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

Laurent Frenzel, Kaan Kavakli, Robert Klamroth, Shyh-Shin Chiou, Amy D. Shapiro, Pengling Sun, Joanne Fuiman, John McKay, Annie F. Fang, Francesca Biondo, Frank Plonski, Jeremy Rupon

Blood (2023) 142 (Supplement 1): 2257.

https://doi.org/10.1182/blood-2023-181223

Published: 2023

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

A Retrospective Cohort Study of Switching Patterns Among Hemophilia a and B Patients Using Optum Electronic Health Records (EHR) Database in the United States (US)

Yun Gu, Kui Huang, Xunming Sun, Xiaofeng Zhou, Rongjun Shen, Lisa J. Young, Amit Chhabra, Gregory Di Russo, Jeremy Rupon, Ian Winburn

Blood (2022) 140 (Supplement 1): 5603–5605.

https://doi.org/10.1182/blood-2022-163569

Published: 2022

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

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

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

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

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

Impact of Treatment Regimen with Moroctocog Alfa (AF-CC) on Bleeding Frequency in Pediatric Aged Subjects with Moderately Severe to Severe Hemophilia A

Mark P. Smith, MD FRACP, FRCPA, Jeremy Rupon, MD, Yasser Wali, MD, Hala Remawi, MD, Joan Korth-Bradley, PharmD, PhD, Lynne Smith, MBA, Pablo Rendo, MD

Blood (2018) 132 (Supplement 1): 1205.

https://doi.org/10.1182/blood-2018-99-111891

Published: 2018

Journal Articles

An Open-Label Study of the Safety and Efficacy of Morotocog Alfa (AF-CC) in Previously Untreated Patients Aged <6 Years in Usual Care Settings

Kaan Kavakli, MD, James Baumann, Francois Pierre Huard, MS, MBA, Lynne Smith, MBA, Christine Alvey, Joan Korth-Bradley, PharmD, PhD, Pablo Rendo, MD, Jeremy Rupon, MD

Blood (2017) 130 (Supplement 1): 1091.

https://doi.org/10.1182/blood.V130.Suppl_1.1091.1091

Published: 2017

NARROW

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 ΑFFINE Gene Therapy Trial

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

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

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

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

A Retrospective Cohort Study of Switching Patterns Among Hemophilia a and B Patients Using Optum Electronic Health Records (EHR) Database in the United States (US)

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

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

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

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

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

Impact of Treatment Regimen with Moroctocog Alfa (AF-CC) on Bleeding Frequency in Pediatric Aged Subjects with Moderately Severe to Severe Hemophilia A

An Open-Label Study of the Safety and Efficacy of Morotocog Alfa (AF-CC) in Previously Untreated Patients Aged <6 Years in Usual Care Settings

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American Society of Hematology

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American Society of Hematology

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