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Journal Articles

A Phase 1/2, First-in-Human, Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia

Suthesh Sivapalaratnam, Steve Austin, Ulrike Lorch, Thomas York, Andrew Want, Ashley Gosnell, Prafull S. Gandhi, Henrik Østergaard, Erik Bjornson, Tara O'Meara, Benny Sorensen, Roger E.G. Schutgens, Catherine Rea
Journal: Blood
Blood (2023) 142 (Supplement 1): 1225.
https://doi.org/10.1182/blood-2023-187279
Published: 2023
Journal Articles

Presenting ADAMTS13 antibody and antigen levels predict prognosis in immune-mediated thrombotic thrombocytopenic purpura

Clinical Trials & Observations
Ferras Alwan, Chiara Vendramin, Karen Vanhoorelbeke, Katy Langley, Vickie McDonald, Steve Austin, Amanda Clark, William Lester, Richard Gooding, Tina Biss, Tina Dutt, Nichola Cooper, Oliver Chapman, Tanya Cranfield, Kenny Douglas, H. G. Watson, J. J. van Veen, Keith Sibson, William Thomas, Lynn Manson, Quentin A. Hill, Sylvia Benjamin, Debra Ellis, John-Paul Westwood, Mari Thomas, Marie Scully
Journal: Blood
Blood (2017) 130 (4): 466–471.
https://doi.org/10.1182/blood-2016-12-758656
Published: 2017
Journal Articles

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors

Margaret V. Ragni, MD, Pencho Georgiev, Tim Mant, Michael Desmond Creagh, Toshko Lissitchkov, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Savita Rangarajan, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, K John Pasi
Journal: Blood
Blood (2016) 128 (22): 2572.
https://doi.org/10.1182/blood.V128.22.2572.2572
Published: 2016
Journal Articles

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

K John Pasi, Pencho Georgiev, Tim Mant, Toshko Lissitchkov, Michael Desmond Creagh, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Margaret V. Ragni, MD, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, Savita Rangarajan
Journal: Blood
Blood (2016) 128 (22): 1397.
https://doi.org/10.1182/blood.V128.22.1397.1397
Published: 2016
Journal Articles

Prospective Evaluation of Bleeding Incidence in Fibrinogen Deficiency (PRO-RBDD Study)

Roberta Palla, PhD, Marzia Menegatti, Marco Boscarino, Jan Blatny, Ondrej Zapletal, Jean-Francois Schved, MD PhD, Muriel Giansily-Blaizot, MD PhD, Susan Halimeh, Britta Lachmann, Helen Platokouki, MD, Helen Pergantou, Simona Maria Siboni, Marlies van Lent, Paul Brons, Britta A.P. Laros-Van Gorkom, Roger E.G. Schutgens, Monique van Haaften-Spoor, Munira Borhany, Naveena Fatima, Danijela Mikovic, Marko Saracevic, Philippe De Moerloose, Alessandro Casini, Nihal Ozdemir, Yilmaz Ay, Steve Austin, Pearl Quartey, Michael Makris, Sarah Brown, Amy D. Shapiro, Adrianna Williamson, John C. Chapin, Fraustina Hsu, Flora Peyvandi, MD PhD
Journal: Blood
Blood (2016) 128 (22): 207.
https://doi.org/10.1182/blood.V128.22.207.207
Published: 2016
Journal Articles

A gain-of-function variant in DIAPH1 causes dominant macrothrombocytopenia and hearing loss

Simon Stritt, Paquita Nurden, Ernest Turro, Daniel Greene, Sjoert B. Jansen, Sarah K. Westbury, Romina Petersen, William J. Astle, Sandrine Marlin, Tadbir K. Bariana, Myrto Kostadima, Claire Lentaigne, Stephanie Maiwald, Sofia Papadia, Anne M. Kelly, Jonathan C. Stephens, Christopher J. Penkett, Sofie Ashford, Salih Tuna, Steve Austin, Tamam Bakchoul, Peter Collins, Rémi Favier, Michele P. Lambert, Mary Mathias, Carolyn M. Millar, Rutendo Mapeta, David J. Perry, Sol Schulman, Ilenia Simeoni, Chantal Thys, BRIDGE-BPD Consortium, Keith Gomez, Wendy N. Erber, Kathleen Stirrups, Augusto Rendon, John R. Bradley, Chris van Geet, F. Lucy Raymond, Michael A. Laffan, Alan T. Nurden, Bernhard Nieswandt, Sylvia Richardson, Kathleen Freson, Willem H. Ouwehand, Andrew D. Mumford
Journal: Blood
Blood (2016) 127 (23): 2903–2914.
https://doi.org/10.1182/blood-2015-10-675629
Published: 2016
Includes: Supplemental data
Journal Articles

A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Weekly and Monthly Dosing Results in Patients with Hemophilia A or B

K John Pasi, Pencho Georgiev, Tim Mant, Michael Desmond Creagh, Toshko Lissitchkov, MD, David Bevan, Steve Austin, Charles R Hay, Brigit Brand, Rashid Kazmi, Pratima Chowdary, Margaret V Ragni, Jihong Chen, Akin Akinc, Benny Sorensen, Savita Rangarajan
Journal: Blood
Blood (2015) 126 (23): 551.
https://doi.org/10.1182/blood.V126.23.551.551
Published: 2015
Journal Articles

Genotype Analysis of Patients with Hereditary Factor X Deficiency Enrolled in 2 Phase 3 Studies of Pdfx, a New High-Purity Factor X Concentrate

Mike Mitchell, PhD, Kaan Kavakli, MD, Miranda Norton, PhD, Steve Austin
Journal: Blood
Blood (2015) 126 (23): 3511.
https://doi.org/10.1182/blood.V126.23.3511.3511
Published: 2015