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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

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

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

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

Journal Articles

Radiologist-Performed Musculoskeletal Ultrasound (MSKUS) for Evaluation of Joint and Soft Tissue Pain Episodes in Patients with Bleeding Disorders

Anjlee Mahajan, MD, Patricia Tobase, PT, DPT, Andrew Phelps, MD, Darcy Phelan, RN, MS, NP, Mary B. Lesh, CNP, MS, NP, Adam Giermasz, MD PhD

Blood (2015) 126 (23): 3266.

https://doi.org/10.1182/blood.V126.23.3266.3266

Published: 2015

Journal Articles

Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy In Adults with Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation.

Adam Giermasz, MD, PhD, Lloyd E. Damon, MD, Lawrence D Kaplan, MD, Willis H. Navarro, MD, Kristen Hege, MD, Charles Linker, MD, Thomas Martin, MD

Blood (2010) 116 (21): 1317.

https://doi.org/10.1182/blood.V116.21.1317.1317

Published: 2010

NARROW

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

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

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

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

Radiologist-Performed Musculoskeletal Ultrasound (MSKUS) for Evaluation of Joint and Soft Tissue Pain Episodes in Patients with Bleeding Disorders

Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy In Adults with Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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