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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

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Steven W. Pipe, MD, Michael Recht, MD PhD, Nigel S. Key, MD, Frank W.G. Leebeek, MD PhD, Giancarlo Castaman, MD, Susan U. Lattimore, R.N., Paul Van Der Valk, Kathelijne Peerlinck, MD PhD, Michiel Coppens, MD, Niamh O'Connell, MD PhD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Peter Kampmann, MD, Karina Meijer, MD PhD, Annette von Drygalski, MD Pharm D, Guy Young, MD, Cedric Hermans, MDMRCP,PhD, Jan Astermark, MD PhD, Robert Klamroth, MD PhD, Richard S. Lemons, MD, Nathan Visweshwar, MD, Shelley Crary, MD MS, Rashid Kazmi, MBBS, Emily Symington, Miguel A. Escobar, MD, Esteban Gomez, MD, Rebecca Kruse-Jarres, MD, Adam Kotowski, MD, Doris Quon, MD PhD, Michael Wang, MD, Allison P. Wheeler, MD, Eileen K Sawyer, PhD, Stephanie Verweij, BSc, Valerie Colletta, MSc, Naghmana Bajma, MD, Robert Gut, MD PhD, Wolfgang A. Miesbach, MD PhD

Blood (2020) 136 (Supplement_2): LBA-6.

https://doi.org/10.1182/blood-2020-143560

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

Assessing Efficacy of Provider and Family Education, Electronic Health Record Documentation Tools, and Information Technology to Increase Use of Hydroxyurea in Children with Sickle Cell Anemia

Kyle Hickman, MD, Steve McCalley, BSN, RN-BC, Vinay Vaidya, MD, Esteban Gomez, MD, Christine M. Knoll, MD, Joanna L. Gendreau, DO, Lori Wagner, MSN, FNP-C, Meenakshi Goyal-Khemka, MD, Brooke Cook, BSN, RN, Kelli Fischbeck, BSN, RN, Sanjay J. Shah, MBBS

Blood (2019) 134 (Supplement_1): 2288.

https://doi.org/10.1182/blood-2019-127770

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

Use of a Electronic Sickle Cell Dashboard to Improve Annual Transcranial Doppler Ultrasound (TCD) Completion Rates

Joanna L. Gendreau, DO, Kelli Fischbeck, BSN, RN, Brooke Cook, BSN, RN, Steve McCalley, BSN, RN-BC, Lori Wagner, MSN, FNP-C, Vinay Vaidya, MD, Meenakshi Goyal-Khemka, MD, Esteban Gomez, MD, Christine M. Knoll, MD, Sanjay J. Shah, MBBS

Blood (2019) 134 (Supplement_1): 3384.

https://doi.org/10.1182/blood-2019-128846

Published: 2019

Journal Articles

Mitochondrial Genome Changes As a Measure Of Iron-Induced Mitochondrial Stress In Transfusion-Dependent Thalassemia

Esteban Gomez, M.D., Cassandra Calloway, Ph.D., Sang Hoon Lee, B.A., Jay Kim, B.A., Navpreet Dhillon, Ginny Gildengorin, PhD, Ashutosh Lal, MD

Blood (2013) 122 (21): 2256.

https://doi.org/10.1182/blood.V122.21.2256.2256

Published: 2013

NARROW

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

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

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

Assessing Efficacy of Provider and Family Education, Electronic Health Record Documentation Tools, and Information Technology to Increase Use of Hydroxyurea in Children with Sickle Cell Anemia

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

Use of a Electronic Sickle Cell Dashboard to Improve Annual Transcranial Doppler Ultrasound (TCD) Completion Rates

Mitochondrial Genome Changes As a Measure Of Iron-Induced Mitochondrial Stress In Transfusion-Dependent Thalassemia

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ASH Publications

American Society of Hematology

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

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

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

Assessing Efficacy of Provider and Family Education, Electronic Health Record Documentation Tools, and Information Technology to Increase Use of Hydroxyurea in Children with Sickle Cell Anemia

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

Use of a Electronic Sickle Cell Dashboard to Improve Annual Transcranial Doppler Ultrasound (TCD) Completion Rates

Mitochondrial Genome Changes As a Measure Of Iron-Induced Mitochondrial Stress In Transfusion-Dependent Thalassemia

Email alerts

ASH Publications

American Society of Hematology

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