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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

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

Frank W.G. Leebeek, MD PhD, Karina Meijer, MD PhD, Michiel Coppens, MD, Peter Kampmann, MD, Robert Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Wolfgang A. Miesbach, MD

Blood (2020) 136 (Supplement 1): 26.

https://doi.org/10.1182/blood-2020-139225

Published: 2020

Journal Articles

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Wolfgang Miesbach, MD PhD, Karina Meijer, Michiel Coppens, MD, Peter Kampmann, MD, Dr. Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 2059.

https://doi.org/10.1182/blood-2019-122535

Published: 2019

Journal Articles

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Frank Leebeek, K Meijer, MD, Michiel Coppens, MD, Peter Kampmann, MD, Robert Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, Erhard Seifried, MD, J Schwaeble, MD, Halvard Bonig, MD, Eileen K Sawyer, PhD, W Miesbach

Blood (2018) 132 (Supplement 1): 3476.

https://doi.org/10.1182/blood-2018-99-109995

Published: 2018

Journal Articles

Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B

Frank W.G. Leebeek, MD PhD, Marco Tangelder, MD PhD, Karina Meijer, MD PhD, Giancarlo Castaman, Federica Cattaneo, MD, Michiel Coppens, MD PhD, Peter Kampmann, MD, Robert Klamroth, Roger E.G. Schutgens, MD PhD, Erhard Seifried, MD PhD, Joachim Schwaeble, MD, Halvard Boenig, MD PhD, Maaike Hendriks, Deya Corzo, MD FAAP, Wolfgang A. Miesbach, MD PhD

Blood (2016) 128 (22): 2314.

https://doi.org/10.1182/blood.V128.22.2314.2314

Published: 2016

Journal Articles

Targeted Whole Exome Sequencing of 87 Predisposition Genes in 156 Patients from the Oresund Region with Bleeding Disorders

Eva B Leinoe, MD PhD, Eva Zetterberg, MD PhD, Olga Oestrup, Molecular biologist, PhD, Peter Kampmann, MD, Norstrom Eva, MD PhD, Nadine G Andersson, MD PhD, Savvas Kinalis, Data scientist, Klaus Qvortrup, Professor MD PhD, Caroline Maria Rossing, MD PhD

Blood (2016) 128 (22): 361.

https://doi.org/10.1182/blood.V128.22.361.361

Published: 2016

NARROW

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

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B

Targeted Whole Exome Sequencing of 87 Predisposition Genes in 156 Patients from the Oresund Region with Bleeding Disorders

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ASH Publications

American Society of Hematology

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

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B

Targeted Whole Exome Sequencing of 87 Predisposition Genes in 156 Patients from the Oresund Region with Bleeding Disorders

Email alerts

ASH Publications

American Society of Hematology

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