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Journal Articles

A Phase 1b Study of ELA026, a Monoclonal Antibody Targeting Signal Regulatory Protein-α/β1/γ, in Patients with Newly Diagnosed and Previously Treated Secondary Hemophagocytic Lymphohistiocytosis

Abhishek Maiti, Naval Daver, Swami P. Iyer, David McCall, Satyen H Gohil, Javier Lopez Jimenez, Hayley Lane, Benjamin Kim, Kim-Hien Dao, Gary Patou, Sandip Panicker, Kelly Covert, Graham Carl Parry, Carl E Allen

Blood (2023) 142 (Supplement 1): 1162.

https://doi.org/10.1182/blood-2023-177795

Published: 2023

Journal Articles

Results from an Association-Scan of the Extended MHC-Class-II Region Using Novel Association-Based Statistical Methods Establish That DQ Allotypes and Race Independently Influence the Risk of Factor VIII Inhibitor Development in Hemophilia-A Patients

Paola Ibarra, Marcio A. Almeida, Vincent P. Diego, Bernadette W. Luu, Kevin R. Viel, Raja Rajalingam, Afshin Ameri, Meera Chitlur, Benjamin Kim, Grecia Castillo, Jerry S. Powell, Juan M. Peralta, Miguel A. Escobar, Sarah Williams-Blangero, John Blangero, Carol K. Kasper, Laura Almasy, Tom E. Howard

Blood (2023) 142 (Supplement 1): 1247.

https://doi.org/10.1182/blood-2023-191140

Published: 2023

Journal Articles

Gene-Centric Association Scans of Pleiotropic Immune-Mediated Disease Genes in the PATH Study Identify Novel Determinants of Factor VIII Inhibitor Risk in Hemophilia-A Patients and Confirm Race as an Independent Predictor

Ruayda Bouls, Marcio A. Almeida, Vincent P. Diego, Bernadette W. Luu, Raja Rajalingam, Afshin Ameri, Kevin R. Viel, Meera Chitlur, Benjamin Kim, Miguel A. Escobar, Sarah Williams-Blangero, Carol K. Kasper, Laura Almasy, John Blangero, Tom E. Howard

Blood (2023) 142 (Supplement 1): 1246.

https://doi.org/10.1182/blood-2023-190094

Published: 2023

Journal Articles

Pre-Clinical Characterization of VGA039, an Anti-Protein S Monoclonal Antibody Being Developed As a Universal Hemostatic Agent for Various Bleeding Disorders

Lilley Leong, Tony Byun, Benjamin Kim, Ian Huck, Abel Silva, Joanne Chan, Young Cho, John Liu, Christopher J. Horvath, Stephen J. Moore, Sandip Panicker

Blood (2022) 140 (Supplement 1): 1666–1667.

https://doi.org/10.1182/blood-2022-170245

Published: 2022

Journal Articles

Trial in Progress: A Phase 1b Study of ELA026 in Patients with Secondary Hemophagocytic Lymphohistiocytosis

Timothy S. Leach, Hayley Lane, Benjamin Kim, Stephen J. Moore, Sandip Panicker, Gary Patou

Blood (2022) 140 (Supplement 1): 8336–8337.

https://doi.org/10.1182/blood-2022-167961

Published: 2022

Journal Articles

Relationship between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Blood (2021) 138 (Supplement 1): 3972.

https://doi.org/10.1182/blood-2021-144508

Published: 2021

Journal Articles

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Blood (2021) 138 (Supplement 1): 4916.

https://doi.org/10.1182/blood-2021-146021

Published: 2021

Journal Articles

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

Steffen Rosen, Stefan Tiefenbacher, Mary Robinson, Mei Huang, Jaydeep Srimani, Donnie Mackenzie, Terri Christianson, K. John Pasi, Savita Rangarajan, Emily Symington, Adam Giermasz, Glenn F. Pierce, Benjamin Kim, Stephen J. Zoog, Christian Vettermann

Blood (2020) 136 (22): 2524–2534.

https://doi.org/10.1182/blood.2020005683

Published: 2020

Includes: Supplemental data

Journal Articles

Vector Shedding and Blood Biodistribution in Patients with Severe Hemophilia a Following Administration of Valoctocogene Roxaparvovec

Annie Clark, PhDPharmD, Kevin Hammon, Krystal Sandza, Richard Torres, Elli Koziol, Jennifer Holcomb, Benjamin Kim, MD, Kala Jayaram, MD, Chris Russell, PhD, Christian Vettermann, PhD, Joshua Henshaw, PhD

Blood (2020) 136 (Supplement 1): 21.

https://doi.org/10.1182/blood-2020-136459

Published: 2020

Journal Articles

Achievement of Normal Circulating Factor VIII Activity Following Bmn 270 AAV5-FVIII Gene Transfer: Interim, Long-Term Efficacy and Safety Results from a Phase 1/2 Study in Patients with Severe Hemophilia a

K. John Pasi, Savita Rangarajan, Benjamin Kim, Will Lester, David Perry, Bella Madan, Fatemeh Tavakkoli, Ke Yang, Glenn F. Pierce, Wing Yen Wong

Blood (2017) 130 (Supplement 1): 603.

https://doi.org/10.1182/blood.V130.Suppl_1.603.603

Published: 2017

Journal Articles

Interim Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in a Phase-1/2 Clinical Study of Bmn 270, an AAV5-Mediated Gene Therapy for Hemophilia a

Brian Long, PhD MS, BSMedTech, Benjamin Kim, Wing Yen Wong, Ke Yang, Christian Vettermann, Nancy Pryer, Romain Hardet, Klaudia Kuranda, Philippe Veron, Federico Mingozzi, Glenn F. Pierce, Becky Schweighardt

Blood (2017) 130 (Supplement 1): 4611.

https://doi.org/10.1182/blood.V130.Suppl_1.4611.4611

Published: 2017

Journal Articles

The Spectrum of Amino Acid Substitutions Resulting from Single Nucleotide Substitutions in the Coagulation Biosystem: Impact on Identification By Mass Spectrometry

Kevin R. Viel, PhD, Benjamin Kim, MD MPhil, Maria Elizabeth Tejero, PhD, Shelley S. Cole, PhD, Tom E. Howard, MD PhD, Amparo Santamaría Ortiz, MD, PhD

Blood (2014) 124 (21): 4221.

https://doi.org/10.1182/blood.V124.21.4221.4221

Published: 2014

Journal Articles

Regional Differences in Patient-Reported Outcomes in a Study of Adult Prophylaxis Versus on-Demand Treatment with Bayer’s Sucrose-Formulated Recombinant Factor VIII: 3-Year Data from Spinart

Jennifer Pocoski, PharmD, Benjamin Kim, MD MPhil, Thomas J. Humphries, MD, Walter Hong

Blood (2014) 124 (21): 1518.

https://doi.org/10.1182/blood.V124.21.1518.1518

Published: 2014

Journal Articles

Assessment of Algorithms to Identify Patients with Thrombophilia Following Venous Thromboembolism

Wendy Hsiao, BS, Thomas Delate, PhD MS, Daniel M Witt, PharmD, Melissa R Meyer, PharmD, Alan Go, MD, Margaret Fang, MD MPH, Benjamin Kim, MD MPhil

Blood (2014) 124 (21): 3513.

https://doi.org/10.1182/blood.V124.21.3513.3513

Published: 2014

Journal Articles

Single-Nucleotide Variations Defining Previously Unreported ADAMTS13 Haplotypes Are Associated With Differential Expression and Activity Of The VWF-Cleaving Protease In a Salvadoran Congenital Thrombotic Thrombocytopenic Purpura Family

Benjamin Kim, M.D., M.Phil., Zachary A. Hing, Andrew Wu, Tal Schiller, Evi B. Struble, Ph.D., David Liuwantara, Ph.D., Pamela H. Kempert, M.D., Eric J. Broxham, M.D., Nathan C. Edwards, Victor J. Marder, M.D., Vijaya L. Simhadri, Ph.D., Zuben E. Sauna, Ph.D., Tom E. Howard, M.D., Ph.D., Chava Kimchi-Sarfaty, Ph.D.

Blood (2013) 122 (21): 2319.

https://doi.org/10.1182/blood.V122.21.2319.2319

Published: 2013

Journal Articles

F8 and HLA -II Haplotypes in the Hispanic Population: Implications for Inhibitor Risk Development in Hispanic Hemophilia A Patients

Benjamin Kim, M.D., M.Phil., Zuben E. Sauna, Ph.D., Melanie A. Carless, Ph.D., Joanne E. Curran, Ph.D., Kevin R. Viel, Ph.D., Shelley S. Cole, Ph.D., Rajalingam Raja, Ph.D., John Blangero, Ph.D., Tom E. Howard, M.D., Ph.D.

Blood (2012) 120 (21): 3365.

https://doi.org/10.1182/blood.V120.21.3365.3365

Published: 2012

Journal Articles

Common SNPs within or near Three Immune Response Genes Implicated in the Risk of FVIII Immunogenicity in Hemophilia A Do Not Influence Steady-State Levels of Their Encoded mRNAs

Tom E. Howard, M.D., Ph.D., Eugene Drigalenko, Ph.D., Matthew P. Johnson, Ph.D., Shelley S. Cole, Ph.D., Benjamin Kim, M.D., M.Phil., Kevin R. Viel, Ph.D., Zuben E. Sauna, Ph.D., Joanne E. Curran, Ph.D., John Blangero, Ph.D., Laura A. Almasy, Ph.D., Harald H. Göring, Ph.D.

Blood (2012) 120 (21): 3366.

https://doi.org/10.1182/blood.V120.21.3366.3366

Published: 2012

Journal Articles

No Evidence of Cis -Acting SNPs In Immune Response Genes Previously Associated with Factor VIII Inhibitors: The San Antonio Family Heart Study

Tom E. Howard, M.D., Ph.D., Kevin R. Viel, Ph.D., Eugene Drigalenko, Ph.D., Shelley Cole, Ph.D., Melinda Epstein, Ph.D., Melanie Carless, Ph.D., Richard Hughes, Ph.D., Benjamin Kim, MD, Tom Dyer, M.S., Kathleen P. Pratt, PhD, Jean W. MacCluer, Ph.D., Joanne E. Curran, Ph.D., Matthew P. Johnson, Ph.D., Eric K. Moses, Ph.D., John Blangero, Ph.D., Laura Almasy, Ph.D., Harald Göring, Ph.D.

Blood (2010) 116 (21): 4422.

https://doi.org/10.1182/blood.V116.21.4422.4422

Published: 2010

Journal Articles

The Role of Immune-Response Gene Variants in Inhibitor Development in Black Patients with Hemophilia A.

Tom E. Howard, MD, PhD, Shelley A. Cole, PhD, Carol K. Kasper, MD, Afshin Ameri, MD, Kavita Natarajan, MD, Raymond G. Watts, MD, Valrie A. Manard, RN, Shelley M. Nakaya, Christine L Kempton, MD, MSc, Martina Kennedy, DO, Benjamin Kim, MD, Rathi V. Iyer, MD, Laura Almasy, PhD

Blood (2009) 114 (22): 1301.

https://doi.org/10.1182/blood.V114.22.1301.1301

Published: 2009

Journal Articles

Erythropoiesis Stimulating Agent (ESA) Use for Anemic Cancer and Chronic Kidney Disease (CKD) Patients in 2008: Advocacy for Conservative Use

Charles L. Bennett, MD, PhD, MPP, Benjamin Kim, MD, Athena T Samaras, BA, Allen R Nissenson, MD, FACP, A. Oliver Sartor, MD, D. Mark Courtney, MD, Robert I. Liem, MD, Dennis P West, PhD, Simone N Boyle, BA, Robin L Wagner, Carolyn E Goldstein, Steven M Trifilio, RPh, David M Spiegel, MD, FACP, Stephen Y Lai, MD, PhD

Blood (2008) 112 (11): 4680.

https://doi.org/10.1182/blood.V112.11.4680.4680

Published: 2008

NARROW

A Phase 1b Study of ELA026, a Monoclonal Antibody Targeting Signal Regulatory Protein-α/β1/γ, in Patients with Newly Diagnosed and Previously Treated Secondary Hemophagocytic Lymphohistiocytosis

Results from an Association-Scan of the Extended MHC-Class-II Region Using Novel Association-Based Statistical Methods Establish That DQ Allotypes and Race Independently Influence the Risk of Factor VIII Inhibitor Development in Hemophilia-A Patients

Gene-Centric Association Scans of Pleiotropic Immune-Mediated Disease Genes in the PATH Study Identify Novel Determinants of Factor VIII Inhibitor Risk in Hemophilia-A Patients and Confirm Race as an Independent Predictor

Pre-Clinical Characterization of VGA039, an Anti-Protein S Monoclonal Antibody Being Developed As a Universal Hemostatic Agent for Various Bleeding Disorders

Trial in Progress: A Phase 1b Study of ELA026 in Patients with Secondary Hemophagocytic Lymphohistiocytosis

Relationship between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

Vector Shedding and Blood Biodistribution in Patients with Severe Hemophilia a Following Administration of Valoctocogene Roxaparvovec

Achievement of Normal Circulating Factor VIII Activity Following Bmn 270 AAV5-FVIII Gene Transfer: Interim, Long-Term Efficacy and Safety Results from a Phase 1/2 Study in Patients with Severe Hemophilia a

Interim Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in a Phase-1/2 Clinical Study of Bmn 270, an AAV5-Mediated Gene Therapy for Hemophilia a

The Spectrum of Amino Acid Substitutions Resulting from Single Nucleotide Substitutions in the Coagulation Biosystem: Impact on Identification By Mass Spectrometry

Regional Differences in Patient-Reported Outcomes in a Study of Adult Prophylaxis Versus on-Demand Treatment with Bayer’s Sucrose-Formulated Recombinant Factor VIII: 3-Year Data from Spinart

Assessment of Algorithms to Identify Patients with Thrombophilia Following Venous Thromboembolism

Single-Nucleotide Variations Defining Previously Unreported ADAMTS13 Haplotypes Are Associated With Differential Expression and Activity Of The VWF-Cleaving Protease In a Salvadoran Congenital Thrombotic Thrombocytopenic Purpura Family

F8 and HLA -II Haplotypes in the Hispanic Population: Implications for Inhibitor Risk Development in Hispanic Hemophilia A Patients

Common SNPs within or near Three Immune Response Genes Implicated in the Risk of FVIII Immunogenicity in Hemophilia A Do Not Influence Steady-State Levels of Their Encoded mRNAs

No Evidence of Cis -Acting SNPs In Immune Response Genes Previously Associated with Factor VIII Inhibitors: The San Antonio Family Heart Study

The Role of Immune-Response Gene Variants in Inhibitor Development in Black Patients with Hemophilia A.

Erythropoiesis Stimulating Agent (ESA) Use for Anemic Cancer and Chronic Kidney Disease (CKD) Patients in 2008: Advocacy for Conservative Use

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