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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

Acquired Factor X Deficiency in Systemic Light Chain Amyloidosis: Factor X Recovery after Bleeding Complications, Splenectomy, Chemotherapy and Remission

Rosa Toenges, MD, Juliane Steinmann, MD, Annelie Siegemund, MD, Hubert Serve, Wolfgang A. Miesbach, MD PhD

Blood (2017) 130 (Supplement 1): 4892.

https://doi.org/10.1182/blood.V130.Suppl_1.4892.4892

Published: 2017

Journal Articles

Factor VIII Activity and Risk of Bleeding in Acquired Hemophilia a: Results from the Gth-AH 01/2010 Study

Katharina Holstein, MD, Andrea Smith, Paul Knöbl, MD PhD, Christiane Dobbelstein, MD, Wolfgang A. Miesbach, MD PhD, Michael Spannagl, MD PhD, Jürgen Heinz, MD PhD, Ute Scholz, MD, Hermann Eichler, MD, Angela Huth-Kühne, MD, Robert Klamroth, MD PhD, Armin Koch, MD PhD, Andreas Tiede, MD

Blood (2017) 130 (Supplement 1): 22.

https://doi.org/10.1182/blood.V130.Suppl_1.22.22

Published: 2017

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

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

Perception of Actual Factor Concentrates and Expectations Towards Future Treatment with New Long-Lasting Products - Results of a Survey Among Haemophilia Patients in Germany - Analysis of Open Questions

Werner Kalnins, Wolfgang A. Miesbach, MD PhD, Sylvia von Mackensen, PhD

Blood (2015) 126 (23): 3269.

https://doi.org/10.1182/blood.V126.23.3269.3269

Published: 2015

Journal Articles

Phase 1-2 Clinical Trial of a Recombinant AAV5 Vector Containing the Human FIX Gene in Patients with Severe or Moderately Severe Haemophilia B

Wolfgang A. Miesbach, MD PhD, Christian Meyer, MD PhD, Bart Nijmeijer, PhD, Florence Salmon, PhD, Nadina Grosios, PhD, Harald Petry, PhD, Frank W.G. Leebeek, MD PhD

Blood (2014) 124 (21): 5948.

https://doi.org/10.1182/blood.V124.21.5948.5948

Published: 2014

Journal Articles

Final Evaluation Of a Prospective, Non-Interventional Study On Efficacy and Tolerability Of a New Generation VWF/FVIII Concentrate In The Treatment Of Von Willebrand Disease

Sonja Alesci, MD, Mario Von Depka, Jennifer Feddern, Susan Halimeh, MD, Oliver Hegener, PhD, Janos Kadar, Wolfgang A. Miesbach, MD, PhD, Ulrike Nowak-Göttl, Inge Scharrer, MD, PhD

Blood (2013) 122 (21): 4775.

https://doi.org/10.1182/blood.V122.21.4775.4775

Published: 2013

Journal Articles

Prognostic Parameters For Remission Of and Survival In Acquired Hemophilia A: Results Of The GTH-AH 01/2010 Multicenter Study

Andreas Tiede, MD, PhD, Jan-Malte Blumtritt, Robert Klamroth, MD, PhD, Saskia Gottstein, MD, Katharina Holstein, MD, Jürgen Heinz, MD, Rüdiger E. Scharf, MD, PhD, Angela Huth-Kühne, MD, PhD, Joachim F. Schenk, MD, PhD, Richard Greil, MD, Wolfgang A. Miesbach, MD, PhD, Ralf Ulrich Trappe, MD, PhD, Ute Scholz, MD, Daniela Manner, MD, Peter Neumeister, MD, Charis von Auer-Wegener, MD, Ulrich Kalus, MD, Klaus Leimkühler, MD, Paul Knoebl, MD

Blood (2013) 122 (21): 205.

https://doi.org/10.1182/blood.V122.21.205.205

Published: 2013

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

Acquired Factor X Deficiency in Systemic Light Chain Amyloidosis: Factor X Recovery after Bleeding Complications, Splenectomy, Chemotherapy and Remission

Factor VIII Activity and Risk of Bleeding in Acquired Hemophilia a: Results from the Gth-AH 01/2010 Study

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

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

Perception of Actual Factor Concentrates and Expectations Towards Future Treatment with New Long-Lasting Products - Results of a Survey Among Haemophilia Patients in Germany - Analysis of Open Questions

Phase 1-2 Clinical Trial of a Recombinant AAV5 Vector Containing the Human FIX Gene in Patients with Severe or Moderately Severe Haemophilia B

Final Evaluation Of a Prospective, Non-Interventional Study On Efficacy and Tolerability Of a New Generation VWF/FVIII Concentrate In The Treatment Of Von Willebrand Disease

Prognostic Parameters For Remission Of and Survival In Acquired Hemophilia A: Results Of The GTH-AH 01/2010 Multicenter Study

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American Society of Hematology

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ASH Publications

American Society of Hematology

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