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Journal Articles

Mim8 Prophylaxis Beyond Bleeding: Investigating Multifaceted, Patient-Reported Outcomes for Hemophilia A in the FRONTIER2 Study

Cedric Hermans, Cihan Ay, Carmen Escuriola, Peter Kampmann, Rikke Medom Meldgaard, Ilgiz Rakhmatullin, Ciprian Tomuleasa, Lize Fie Ditteke van Vulpen, Michael Wang, Irena Woźnica-Karczmarz, Sophie Susen

Blood (2024) 144 (Supplement 1): 1212.

https://doi.org/10.1182/blood-2024-198395

Published: 2024

Journal Articles

Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B

Frank W.G. Leebeek, Karina Meijer, Michiel Coppens, Peter Kampmann, Dr. Klamroth, Paul van der Valk, Paul E. Monahan, Karen Pinachyan, Sandra LeQuellec, Wolfgang Miesbach

Blood (2024) 144 (Supplement 1): 3578.

https://doi.org/10.1182/blood-2024-198379

Published: 2024

Journal Articles

Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial

Blood (2024) 143 (22): 2256–2269.

https://doi.org/10.1182/blood.2023021864

Published: 2024

Includes: Supplemental data

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

Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B

Blood (2023) 142 (Supplement 1): 1055.

https://doi.org/10.1182/blood-2023-187624

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

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

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Wolfgang Miesbach, Karina Meijer, Michiel Coppens, Peter Kampmann, Robert Klamroth, Roger Schutgens, Marco Tangelder, Giancarlo Castaman, Joachim Schwäble, Halvard Bonig, Erhard Seifried, Federica Cattaneo, Christian Meyer, Frank W. G. Leebeek

Blood (2018) 131 (9): 1022–1031.

https://doi.org/10.1182/blood-2017-09-804419

Published: 2018

Includes: Supplemental data

Journal Articles

Stable Elevations in FIX Activity and Reductions in Annualized Bleeding Rate over up to 2 Years of Follow-up of Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Frank W.G. Leebeek, MD PhD, K Meijer, Michiel Coppens, MD PhD, Peter Kampmann, R Klamroth, Roger Schutgens, MD PhD, Giancarlo Castaman, Erhard Seifried, MD PhD, J Schwaeble, Halvard Bonig, MD MA, Eileen K Sawyer, Wolfgang A. Miesbach, MD PhD

Blood (2017) 130 (Supplement 1): 602.

https://doi.org/10.1182/blood.V130.Suppl_1.602.602

Published: 2017

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

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

NARROW

Mim8 Prophylaxis Beyond Bleeding: Investigating Multifaceted, Patient-Reported Outcomes for Hemophilia A in the FRONTIER2 Study

Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B

Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial

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

Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B

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

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Stable Elevations in FIX Activity and Reductions in Annualized Bleeding Rate over up to 2 Years of Follow-up of Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

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

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

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American Society of Hematology

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American Society of Hematology

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