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Journal Articles

Safety and Efficacy of Mim8 Prophylaxis Administered Once Every Two Weeks for Patients with Hemophilia A with or without Inhibitors: Interim Analysis of the FRONTIER4 Open-Label Extension Study

Tadashi Matsushita, Pratima Chowdary, Atanas Mitkov Banchev, Kaan Kavakli, Johanna A. Kremer Hovinga, Jerzy Windyga, Víctor Jiménez-Yuste, Julien Bovet, Llenalia María García Fernández, Guy Young

Blood (2024) 144 (Supplement 1): 718.

https://doi.org/10.1182/blood-2024-198617

Published: 2024

Journal Articles

Reduced Doses of Factor Concentrates and Bypassing Agents to Treat Breakthrough Bleeds in Patients with Hemophilia A and B on Fitusiran Antithrombin-Based Dosing Regimen: ATLAS-OLE

Steven W. Pipe, Kaan Kavakli, Tadashi Matsushita, Huyen Tran, Bulent Zulfikar, Laurel Menapace, Marja Puurunen, Wenruo Hu, Yuqian Shen, Chanchala Kaddi, Vanessa Salinas

Blood (2024) 144 (Supplement 1): 128.

https://doi.org/10.1182/blood-2024-206946

Published: 2024

Journal Articles

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 ΑFFINE Gene Therapy Trial

Blood (2024) 144 (Supplement 1): 1053.

https://doi.org/10.1182/blood-2024-208486

Published: 2024

Journal Articles

Results from the Hemophilia Α Cohort of the Non-Investigational Lead-in Study: Prospective Collection of Bleeding Rate in Participants with Hemophilia Α Prior to Phase 3 Study (AFFINE) of Giroctocogene Fitelparvovec

Laurent Frenzel, Kaan Kavakli, Andrew D. Leavitt, Ali Bülent Antmen, Davide Matino, Margareth Ozelo, Hazza Alzahrani, Barbara A. Konkle, Maria de los Angeles Resa, Annie F. Fang, Jasmine Healy, Delphine Agathon, Francesca Biondo, Frank Plonski

Blood (2024) 144 (Supplement 1): 3575.

https://doi.org/10.1182/blood-2024-193368

Published: 2024

Journal Articles

Use of Fidanacogene Elaparvovec, a Gene Therapy Vector, to Deliver a Stable, Fully Functional Human Factor IX Transgene for the Treatment of Hemophilia B: A Combined Analysis of Safety

Ben Samelson-Jones, Laurent Frenzel, Kaan Kavakli, Adam Cuker, Jerome M. Teitel, Pengling Sun, Lisa J Wilcox, Francesca Biondo, Benjamin Hutter, John McKay, Delphine Agathon, Frank Plonski

Blood (2024) 144 (Supplement 1): 3577.

https://doi.org/10.1182/blood-2024-193453

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

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

Steven W. Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Sonia Deb, Markus Niggli, Tiffany Chang, Michaela Lehle, Karin Fijnvandraat

Blood (2024) 143 (14): 1355–1364.

https://doi.org/10.1182/blood.2023021832

Published: 2024

Includes: Supplemental data

Journal Articles

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

Laurent Frenzel, Kaan Kavakli, Robert Klamroth, Shyh-Shin Chiou, Amy D. Shapiro, Pengling Sun, Joanne Fuiman, John McKay, Annie F. Fang, Francesca Biondo, Frank Plonski, Jeremy Rupon

Blood (2023) 142 (Supplement 1): 2257.

https://doi.org/10.1182/blood-2023-181223

Published: 2023

Journal Articles

Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study

Steven Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Tiffany Chang, Michaela Lehle, Markus Niggli, Karin Fijnvandraat

Blood (2023) 142 (Supplement 1): 505.

https://doi.org/10.1182/blood-2023-177963

Published: 2023

Journal Articles

Fitusiran Prophylaxis Improves Health-Related Quality of Life in People with Hemophilia a or B, with or without Inhibitors: Results of ATLAS-PPX Study

Gili Kenet, Beatrice Nolan, Tadashi Matsushita, Guy Young, Ting Quan, Viridiana Cano, Christian Sussebach, Shauna Andersson, Baisong Mei, Marion Afonso, Kaan Kavakli

Blood (2022) 140 (Supplement 1): 7977–7978.

https://doi.org/10.1182/blood-2022-167169

Published: 2022

Journal Articles

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Alok Srivastava, Savita Rangarajan, Kaan Kavakli, Robert Klamroth, Gili Kenet, Liane Khoo, Chur-Woo You, Weiqun Xu, Niel Malan, Laurent Frenzel, Catherine N. Bagot, Oleksandra Stasyshyn, Chia-Yau Chang, Stacey Poloskey, Shauna Andersson, Zhiying Qiu, Baisong Mei, Steven W. Pipe

Blood (2021) 138 (Supplement 2): LBA-3.

https://doi.org/10.1182/blood-2021-155018

Published: 2021

Journal Articles

Recombinant Porcine Factor VIII in Patients with Congenital Hemophilia a with Inhibitors Undergoing Surgery: Phase 3, Multicenter, Single Arm, Open-Label Study

Christian Pfrepper, Paolo Radossi, Jerzy Windyga, Kaan Kavakli, Roger E.G. Schutgens, Nazan Sarper, Jovanna Baptista, Kayode Badejo, Bruce Ewenstein, Nisha Jain

Blood (2021) 138 (Supplement 1): 2109.

https://doi.org/10.1182/blood-2021-146179

Published: 2021

Journal Articles

Surgeries and Diagnostic Procedures in Hemophilia Patients on Concizumab Prophylaxis: Results from the Phase 2 Explorer4 and Explorer5 Trials

Allison P. Wheeler, Gary Benson, Hermann Eichler, Sidsel Marie Tønder, Katarina Cepo, Victor Jimenez Yuste, Kaan Kavakli, Lily LL Wong, Tadashi Matsushita

Blood (2021) 138 (Supplement 1): 345.

https://doi.org/10.1182/blood-2021-148571

Published: 2021

Journal Articles

Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)

Guy Young, Alok Srivastava, Kaan Kavakli, Cecil Ross, Jameela Sathar, Huyen Tran, Runhui Wu, Jing Sun, Stacey Poloskey, Zhiying Qui, Salim Kichou, Shauna R. Andersson, Baisong Mei, Savita Rangarajan

Blood (2021) 138 (Supplement 1): 4.

https://doi.org/10.1182/blood-2021-150273

Published: 2021

Journal Articles

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results

Amy D. Shapiro, Pantep Angchaisuksiri, Jan Astermark, Gary Benson, Giancarlo Castaman, Pratima Chowdary, Hermann Eichler, Victor Jiménez-Yuste, Kaan Kavakli, Tadashi Matsushita, Lone Hvitfeldt Poulsen, Allison P. Wheeler, Guy Young, Silva Zupancic-Salek, Johannes Oldenburg

Blood (2019) 134 (22): 1973–1982.

https://doi.org/10.1182/blood.2019001542

Published: 2019

Includes: Multimedia, Supplemental data

Journal Articles

An Update on rFVIIa Use in Females with Rare Bleeding Disorders

Meera B. Chitlur, MD, Manuel Carcao, David Cooper, Shilpa Jain, Kaan Kavakli, Bryce Kerlin, Skye Peltier, Man-Chiu Poon, Hossam Saad, Amy Shapiro

Blood (2019) 134 (Supplement_1): 1119.

https://doi.org/10.1182/blood-2019-130298

Published: 2019

Journal Articles

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Severe Hemophilia a without Inhibitors: Results from the Phase 2 explorer5 Trial

Hermann Eichler, Pantep Angchaisuksiri, MD, Jan Astermark, MD PhD, Gary Benson, Katarina Cepo, Pratima Chowdary, MD, Ute Friedrich, PhD, Kaan Kavakli, Allison P Wheeler, MD, Johannes Oldenburg, MD PhD

Blood (2019) 134 (Supplement_1): 2417.

https://doi.org/10.1182/blood-2019-122300

Published: 2019

Journal Articles

An Open-Label Study of the Safety and Efficacy of Morotocog Alfa (AF-CC) in Previously Untreated Patients Aged <6 Years in Usual Care Settings

Kaan Kavakli, MD, James Baumann, Francois Pierre Huard, MS, MBA, Lynne Smith, MBA, Christine Alvey, Joan Korth-Bradley, PharmD, PhD, Pablo Rendo, MD, Jeremy Rupon, MD

Blood (2017) 130 (Supplement 1): 1091.

https://doi.org/10.1182/blood.V130.Suppl_1.1091.1091

Published: 2017

Journal Articles

Concizumab (Anti-TFPI) Exposure-Response Modeling in Patients with Hemophilia A

Hermann Eichler, MD, Pantep Angchaisuksiri, MD, Kaan Kavakli, MD, Paul Knöbl, MD PhD, Jerzy Windyga, Victor Jimenez-Yuste, Ute Friedrich, PhD, Philip Delff Andersen, Pratima Chowdary

Blood (2017) 130 (Supplement 1): 3672.

https://doi.org/10.1182/blood.V130.Suppl_1.3672.3672

Published: 2017

Journal Articles

A Multicenter, Retrospective Data Collection Study on the Compassionate Use of a Plasma-Derived Factor X Concentrate to Treat Patients with Hereditary Factor X Deficiency

James N. Huang, MD, Ri Liesner, Chioma Akenezi, BSc, PgDip, Steven K. Austin, BMedSci, MBBS, Kaan Kavakli, MD

Blood (2017) 130 (Supplement 1): 3736.

https://doi.org/10.1182/blood.V130.Suppl_1.3736.3736

Published: 2017

NARROW

Safety and Efficacy of Mim8 Prophylaxis Administered Once Every Two Weeks for Patients with Hemophilia A with or without Inhibitors: Interim Analysis of the FRONTIER4 Open-Label Extension Study

Reduced Doses of Factor Concentrates and Bypassing Agents to Treat Breakthrough Bleeds in Patients with Hemophilia A and B on Fitusiran Antithrombin-Based Dosing Regimen: ATLAS-OLE

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 ΑFFINE Gene Therapy Trial

Results from the Hemophilia Α Cohort of the Non-Investigational Lead-in Study: Prospective Collection of Bleeding Rate in Participants with Hemophilia Α Prior to Phase 3 Study (AFFINE) of Giroctocogene Fitelparvovec

Use of Fidanacogene Elaparvovec, a Gene Therapy Vector, to Deliver a Stable, Fully Functional Human Factor IX Transgene for the Treatment of Hemophilia B: A Combined Analysis of Safety

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

Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study

Fitusiran Prophylaxis Improves Health-Related Quality of Life in People with Hemophilia a or B, with or without Inhibitors: Results of ATLAS-PPX Study

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Recombinant Porcine Factor VIII in Patients with Congenital Hemophilia a with Inhibitors Undergoing Surgery: Phase 3, Multicenter, Single Arm, Open-Label Study

Surgeries and Diagnostic Procedures in Hemophilia Patients on Concizumab Prophylaxis: Results from the Phase 2 Explorer4 and Explorer5 Trials

Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results

An Update on rFVIIa Use in Females with Rare Bleeding Disorders

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Severe Hemophilia a without Inhibitors: Results from the Phase 2 explorer5 Trial

An Open-Label Study of the Safety and Efficacy of Morotocog Alfa (AF-CC) in Previously Untreated Patients Aged <6 Years in Usual Care Settings

Concizumab (Anti-TFPI) Exposure-Response Modeling in Patients with Hemophilia A

A Multicenter, Retrospective Data Collection Study on the Compassionate Use of a Plasma-Derived Factor X Concentrate to Treat Patients with Hereditary Factor X Deficiency

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American Society of Hematology

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American Society of Hematology

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