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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

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Claude Négrier, Margaret V. Ragni, MDMPH, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Steven W. Pipe, MD, Gili Kenet, Savita Rangarajan, MBBS,MDFRCP,FRCPath, Salim Kichou, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 36–37.

https://doi.org/10.1182/blood-2020-136536

Published: 2020

Journal Articles

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Steven W. Pipe, MD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Toshko Lissitchkov, Margaret V. Ragni, MDMPH, Claude Négrier, Qifeng Yu, Stacey Poloskey, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-136501

Published: 2020

Journal Articles

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

Amy D Shapiro, MD, Barbara A. Konkle, MD, Stacy E. Croteau, MD, Wolfgang A. Miesbach, MD, Charles Richard Morris Hay, MDFRCP,FRCPath, Rashid Kazmi, MBBS, Marina Mihova, Savita Rangarajan, MD MBBS, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2020) 136 (Supplement 1): 8.

https://doi.org/10.1182/blood-2020-141381

Published: 2020

Journal Articles

Examination and Validation of a Patient-Centric Joint Metric: "Problem Joint"; Empirical Evidence from the CHESS US Dataset

Tom Burke, Idaira Rodriguez Santana, PhD, Pratima Chowdary, MD MBBS, MRCP, FRCPATH, Randall Curtis, MBA, Kate Khair, PhD, Michael Laffan, DM, FRCP, FRCPath, Paul McLaughlin, MSc, MMACP, Declan Noone, MSc, Brian O'Mahony, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Mark W Skinner, JD, Jamie O'Hara, MSc

Blood (2020) 136 (Supplement 1): 25–26.

https://doi.org/10.1182/blood-2020-140950

Published: 2020

Journal Articles

Thrombosis and Risk Management in Paediatric Inflammatory Multisystem Syndrome - Temporally Associated with Sars-CoV2 (PIMS-TS)

Ho Pui Jeff Lam, Jayanthi Alamelu, James Brighouse, MBBS FRPCH, Owen Miller, FRCPCH, Jonathan Lillie, PRCPCH, Baba PD Inusa, MDMBBS

Blood (2020) 136 (Supplement 1): 32–33.

https://doi.org/10.1182/blood-2020-142424

Published: 2020

Journal Articles

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

Rachael F. Grace, MD, D. Mark Layton, MB BS, FRCP, FRCPCH, Frédéric Galactéros, MD PhD, Wilma Barcellini, MD, Eduard J. van Beers, MD PhD, Hassan M. Yaish, MD, Yaddanapudi Ravindranath, MBBS, Kevin H. M. Kuo, MDMSc,FRCPC, Sujit Sheth, MD, Janet L. Kwiatkowski, MD MSCE, Lei Hua, PhD, Peter F. Hawkins, PhD, Chris Mix, MD, Bertil Glader, MD PhD

Blood (2019) 134 (Supplement_1): 3512.

https://doi.org/10.1182/blood-2019-123406

Published: 2019

Journal Articles

ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A

Beatrice Nolan, Johnny Mahlangu, Guy Young, Barbara A Konkle, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Johannes Oldenburg, MD PhD, Keiji Nogami, MDPhD, Nikola Tripkovic, Huixing Yuan, Dan Rudin

Blood (2018) 132 (Supplement 1): 1192.

https://doi.org/10.1182/blood-2018-99-118586

Published: 2018

Journal Articles

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

Margaret Ragni, Roshni Kulkarni, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Kathelijn Fischer, Johnny Mahlangu, Amy Shapiro, Beatrice Nolan, Johannes Oldenburg, Tadashi Matsushita, Annemieke Willemze, Huixing Yuan, Dan Rudin

Blood (2018) 132 (Supplement 1): 1214.

https://doi.org/10.1182/blood-2018-99-110100

Published: 2018

Journal Articles

The Role of Antithrombin Lowering in Patients with Hemophilia: Hemostatic Control Pre- and Post-Fitusiran Dosing Interruption

Margaret V. Ragni, MD MPH, Pencho Georgiev, MD, Michael Desmond Creagh, Toshko Jelev Lissitchkov, MD, Steve K. Austin, BMedSci, MBBS, Charles Richard Morris Hay, MDFRCP, FRCPath, Inga Hegemann, MD, Syed Rashid Saeed Kazmi, MBBS, Pratima Chowdary, MD, Savita Rangarajan, MBBS, MD, Meredith C. Foster, ScD, Qifeng Yu, PhD, Craig Benson, MD MS, Kate Madigan, MD, Huy Van Nguyen, PharmD, Shariq Ali, PhD, Ketaki Kadam, MS, Pallavi Jain, PharmD, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2018) 132 (Supplement 1): 2477.

https://doi.org/10.1182/blood-2018-99-112998

Published: 2018

Journal Articles

Gene Therapy in Hemophilia: An Assessment of Hematologists' Knowledge Gaps and Attitudes

Simi Hurst, PhD, Charlotte Warren, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2018) 132 (Supplement 1): 3485.

https://doi.org/10.1182/blood-2018-99-119116

Published: 2018

Journal Articles

Effects of UGT1A1 Polymorphism, Glucose-6-Phosphate Dehydrogenase Deficiency and Deletional (-3.7кb) α-Thalassemia Among Young Nigerians with Sickle Cell Anemia

Oladele Olatunya, MD, Dulcinéia Martins de Albuquerque, Daniela Pinheiro Leonardo, BS, MSc, Kleber Yotsumoto Fertrin, MD PhD, Adekunle Adekile, MBBS, PhD FMCPaed, FWACP, FRCPCH, FRCP, Fernando Ferreira Costa, MD PhD

Blood (2017) 130 (Supplement 1): 2242.

https://doi.org/10.1182/blood.V130.Suppl_1.2242.2242

Published: 2017

Journal Articles

Red Blood Cells Microparticles Are Associated with Hemolysis Markers and Leg Ulceration in Patients with Sickle Cell Disease

Oladele Olatunya, MD, Carolina Lanaro, PhD, Ana Leda Figueiredo Longhini, PhD, Carla Fernanda Franco-Penteado, BSc, PhD, Sara Teresinha Olalla Saad, MD PhD, Kleber Yotsumoto Fertrin, MD PhD, Adekunle Adekile, MBBS, PhD FMCPaed, FWACP, FRCPCH, FRCP, Fernando Ferreira Costa, MD PhD

Blood (2017) 130 (Supplement 1): 3510.

https://doi.org/10.1182/blood.V130.Suppl_1.3510.3510

Published: 2017

Journal Articles

Exploring Pre-B Cell Receptor Signalling As a Therapeutic Target in Acute Lymphoblastic Leukaemia

Ali Alhammer, M.Sc, Marian Case, MSc, Helen J Blair, PhD, Josef Vormoor, MD FRCPCH, Julie Irving, PhD

Blood (2016) 128 (22): 4066.

https://doi.org/10.1182/blood.V128.22.4066.4066

Published: 2016

Journal Articles

Haemopoietic Stem Cell Transplantation for Diamond Blackfan Anaemia Leads to Early and Sustained Engraftment with Good Long-Term Outcomes, but Has an Increased Risk of Gut Toxicity and Lung GvHD

Farah O'Boyle, PhD, Anne Bradshaw, Richard M Szydlo, Josu de la Fuente, PhD FRCP, FRCPI, FRCPCH, FRCPath

Blood (2016) 128 (22): 2679.

https://doi.org/10.1182/blood.V128.22.2679.2679

Published: 2016

Journal Articles

R4 Rgs Subfamily Proteins Negatively Regulates SDF-1/CXCR4 Signaling in CD34+ Hematopoietic Stem and Progenitor Cells

Kam Tong Leung, PhD, Yorky Tsin Sik Wong, BSc, Karen Li, PhD, Kathy Yuen Yee Chan, PhD, Xiao-Bing Zhang, PhD, Ting Fan Leung, MD, Pak Cheung Ng, MD FRCPCH, Chi Kong Li, MD

Blood (2016) 128 (22): 5048.

https://doi.org/10.1182/blood.V128.22.5048.5048

Published: 2016

Journal Articles

Fludarabine/Treosulfan/Thiotepa/ATG Conditioning for Matched and Single Antigen Mismatched Unrelated Bone Marrow Transplantation in Haemoglobinopathies Is Feasible and Leads to Early and Sustanined Engraftment with No Long-Term Toxicity or GvHD

Farah O'Boyle, PhD, Leena Karnik, MD FRCPath, MBBS,, Anne Bradshaw, Richard M Szydlo, Josu de la Fuente, PhD FRCP, FRCPI, FRCPCH, FRCPath

Blood (2016) 128 (22): 2468.

https://doi.org/10.1182/blood.V128.22.2468.2468

Published: 2016

Journal Articles

Fludarabine/Treosulfan/Thiotepa/ATG Conditioning for Related Transplantation in β Thalassaemia Major Leads to Early and Sustained Engraftment with Low Incidence of VOD and GvHD

Farah O'Boyle, PhD, Leena Karnik, MD FRCPath, MBBS,, Anne Bradshaw, Richard M Szydlo, Josu de la Fuente, PhD FRCP, FRCPI, FRCPCH, FRCPath

Blood (2016) 128 (22): 1283.

https://doi.org/10.1182/blood.V128.22.1283.1283

Published: 2016

Journal Articles

Variable Penetrance Is Linked with Monoallelic Gene Expression in Inherited GATA2 -Mutated MDS/AML

Ahad Al Seraihi, BSc, MSc, Ana Rio-Machin, BSc, MSc, PhD, Kiran Tawana, MD PhD, Csaba Bödör, BSc, PhD, Shamzah Araf, MD, James A Heward, BSc, PhD, Matthew Smith, MBBS, MA, MRCP, MRCPath, MD, Sameena Iqbal, Steven Best, PhD, Nicholas Lea, PhD, Donal McLornan, MB, Bch (Hons), PhD MRCP, FRCPath, Alicia Ellison, BSc, Hemanth Tummala, PhD, Shirleny Romualdo Cardoso, BSc, Jamie D. Cavenagh, Tom Vulliamy, FRCPath, Inderjeet Dokal, MD FRCPCH, FRCPath, FMedSci, Jude Fitzgibbon

Blood (2016) 128 (22): 3916.

https://doi.org/10.1182/blood.V128.22.3916.3916

Published: 2016

Journal Articles

History of Asthma is Associated with Albuminuria within an International Pediatric Cohort of Sickle Cell Disease Patients: A Casire Group Analysis

Immacolata Tartaglione, MD, Baba PD Inusa, MBBS, FMCPaed, FRCPCH, MRCP, DCP, Raffaella Colombatti, MD PhD, Deepa Manwani, MD, Silverio Perrotta, Eugenia Vicky Naa Kwarley Asare, MBChB, BSc, Connie M. Piccone, MD, Fredericka Sey, MB ChB, Charles Antwi-Boasiako, PhD, Laura Sainati, MD, Adetola A. Kassim, MD MS, Donna Boruchov, MD, Angela Rivers, MD PhD, William Zempsky, MD, Crawford John Strunk, MD, Biree Andemariam, MD, Andrew D. Campbell, MD

Blood (2016) 128 (22): 3668.

https://doi.org/10.1182/blood.V128.22.3668.3668

Published: 2016

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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

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

Examination and Validation of a Patient-Centric Joint Metric: "Problem Joint"; Empirical Evidence from the CHESS US Dataset

Thrombosis and Risk Management in Paediatric Inflammatory Multisystem Syndrome - Temporally Associated with Sars-CoV2 (PIMS-TS)

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

The Role of Antithrombin Lowering in Patients with Hemophilia: Hemostatic Control Pre- and Post-Fitusiran Dosing Interruption

Gene Therapy in Hemophilia: An Assessment of Hematologists' Knowledge Gaps and Attitudes

Effects of UGT1A1 Polymorphism, Glucose-6-Phosphate Dehydrogenase Deficiency and Deletional (-3.7кb) α-Thalassemia Among Young Nigerians with Sickle Cell Anemia

Red Blood Cells Microparticles Are Associated with Hemolysis Markers and Leg Ulceration in Patients with Sickle Cell Disease

Exploring Pre-B Cell Receptor Signalling As a Therapeutic Target in Acute Lymphoblastic Leukaemia

Haemopoietic Stem Cell Transplantation for Diamond Blackfan Anaemia Leads to Early and Sustained Engraftment with Good Long-Term Outcomes, but Has an Increased Risk of Gut Toxicity and Lung GvHD

R4 Rgs Subfamily Proteins Negatively Regulates SDF-1/CXCR4 Signaling in CD34+ Hematopoietic Stem and Progenitor Cells

Fludarabine/Treosulfan/Thiotepa/ATG Conditioning for Matched and Single Antigen Mismatched Unrelated Bone Marrow Transplantation in Haemoglobinopathies Is Feasible and Leads to Early and Sustanined Engraftment with No Long-Term Toxicity or GvHD

Fludarabine/Treosulfan/Thiotepa/ATG Conditioning for Related Transplantation in β Thalassaemia Major Leads to Early and Sustained Engraftment with Low Incidence of VOD and GvHD

Variable Penetrance Is Linked with Monoallelic Gene Expression in Inherited GATA2 -Mutated MDS/AML

History of Asthma is Associated with Albuminuria within an International Pediatric Cohort of Sickle Cell Disease Patients: A Casire Group Analysis

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