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Journal Articles

Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study

Andrew D. Leavitt, Barbara A. Konkle, Kimo C. Stine, Nathan Visweshwar, Thomas J. Harrington, Adam Giermasz, Steven Arkin, Annie Fang, Frank Plonski, Anne Yver, Florence Ganne, Delphine Agathon, Maria de los Angeles Resa, Li-Jung Tseng, Gregory Di Russo, Bettina M. Cockroft, Liching Cao, Jeremy Rupon

Blood (2024) 143 (9): 796–806.

https://doi.org/10.1182/blood.2022018971

Published: 2024

Includes: Supplemental data

Journal Articles

Potential Predictive Biomarkers for the Development and Outcomes of Inhibitors in Hemophilia A Patients

Meng-Ni Fan, Tangliang Shen, Xiaohe Cai, Ting-Yen Chao, Marilyn Manco-Johnson, Weidong Xiao, Barbara A. Konkle, Lei Li, Carol H. Miao

Blood (2023) 142 (Supplement 1): 2621.

https://doi.org/10.1182/blood-2023-187660

Published: 2023

Journal Articles

Adherence to Clotting Factor Treatment and Association with Bleeding and Health-Related Quality of Life in Persons with Hemophilia

Duc Quang Tran Jr., Megan M. Ullman, Brenda Riske, Cathy Buranahirun-Burns, Amy D. Shapiro, Margaret V. Ragni, Barbara A. Konkle, Nicole Crook, Jonathan C. Roberts, Randall Curtis, Joanne Wu, Michael B. Nichol

Blood (2023) 142 (Supplement 1): 7334.

https://doi.org/10.1182/blood-2023-187387

Published: 2023

Journal Articles

Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α

Thomas J. Harrington, Adam Giermasz, Nathan Visweshwar, Andrew D. Leavitt, Barbara A. Konkle, Jeremy Rupon, Gregory Dirusso, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie F. Fang, Steven Arkin

Blood (2023) 142 (Supplement 1): 1054.

https://doi.org/10.1182/blood-2023-179422

Published: 2023

Journal Articles

Incorporation of O-Glycopeptides into FVIII Decreases Its Immunogenicity

Meng-Ni Fan, Matthew Chrzanowski, Amber Vander Kooi, Tangliang Shen, Rodney M. Camire, Xiaohe Cai, Ting-Yen Chao, Junping Zhang, Barbara A. Konkle, Carol H. Miao, Lei Li, Weidong Xiao

Blood (2023) 142 (Supplement 1): 1228.

https://doi.org/10.1182/blood-2023-187093

Published: 2023

Journal Articles

How I approach bleeding in hospitalized patients

Bethany Samuelson Bannow, Barbara A. Konkle

Blood (2023) 142 (9): 761–768.

https://doi.org/10.1182/blood.2021014766

Published: 2023

Journal Articles

Long-Read Sequencing Resolves Complex Structural Variants and Identifies Missing Disease-Causing Variants in Unsolved Cases of Hemophilia

Danny E. Miller, Miranda Galey, Shelley N Fletcher, Kerry Lannert, Marsha M Wheeler, Renuka Kandhaya-Pillai, Junko Oshima, Barbara A. Konkle, Evan E Eichler, Jill M. Johnsen

Blood (2022) 140 (Supplement 1): 10716–10717.

https://doi.org/10.1182/blood-2022-169355

Published: 2022

Journal Articles

The burden of heavy menstrual bleeding

Barbara A. Konkle

Blood (2022) 140 (16): 1747–1748.

https://doi.org/10.1182/blood.2022017727

Published: 2022

Journal Articles

Depression and Anxiety in Persons with Von Willebrand Disease

Jonathan C. Roberts, Roshni Kulkarni, Peter A. Kouides, Robert F. Sidonio, Jr., Shannon L Carpenter, Barbara A Konkle, Joanne Wu, Megan M. Ullman, Randall Curtis, Judith Baker, Nicole Crook, Michael B. Nichol

Blood (2021) 138 (Supplement 1): 4052.

https://doi.org/10.1182/blood-2021-151625

Published: 2021

Journal Articles

Efanesoctocog Alfa Half-Life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies

Janice M. Staber, Toshko Lissitchkov, Barbara A. Konkle, Amy D. Shapiro, Doris V. Quon, Roshni Kulkarni, Melinda Hamilton, Ekta Seth Chhabra, Suresh Katragadda, Arman Altincatal, Annemieke Willemze, Jennifer Dumont, Margaret V. Ragni

Blood (2021) 138 (Supplement 1): 1035.

https://doi.org/10.1182/blood-2021-148534

Published: 2021

Journal Articles

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia a

Nathan Visweshwar, Thomas J. Harrington, Andrew D Leavitt, Barbara A. Konkle, Adam Giermasz, Kimo Stine, Jeremy Rupon, Gregory Di Russo, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie Fang, Steven Arkin

Blood (2021) 138 (Supplement 1): 564.

https://doi.org/10.1182/blood-2021-148651

Published: 2021

Journal Articles

Bleeding Data across Baseline FIX Expression Levels in People with Hemophilia B: An Analysis Using the 'Factor Expression Study'

Tom Burke, Anum Shaikh, Talaha Ali, Nanxin Li, Barbara A Konkle, Declan Noone, Brian O'Mahony, Steven W Pipe, Jamie O'Hara

Blood (2021) 138 (Supplement 1): 592.

https://doi.org/10.1182/blood-2021-152551

Published: 2021

Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

A Multi-Omics Approach to X-Chromosome Inactivation (XCI) Identifies Severe Xci Skewing in Female Genetic Carriers of Hemophilia

Marsha M Wheeler, PhD, Kathryn Sheldon, Shelley N Fletcher, Angela Dove, Kerry W Lannert, Chris Frazar, Barbara A. Konkle, MD, Deborah A Nickerson, Jill M Johnsen, MD

Blood (2020) 136 (Supplement 1): 19–20.

https://doi.org/10.1182/blood-2020-143066

Published: 2020

Journal Articles

Healthcare Utilization and Health Related Quality of Life in Persons with Von Willebrand Disease

Jonathan C. Roberts, MD, Roshni Kulkarni, MD, Peter A. Kouides, MD, Robert F. Sidonio, Jr., MD, Shannon L Carpenter, MD, Barbara A. Konkle, MD, Joanne Wu, MDMS, Judith Baker, DrPH, MHSA, Megan M. Ullman, MA, MPH, Randall Curtis, MBA, Steven Carrasco, MPH, Duc Quang Tran, Jr., MD, Michael B. Nichol, PhD

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-143055

Published: 2020

Journal Articles

Results of Genetic Analysis of 11,341 Participants Enrolled in the My Life, Our Future (MLOF) Hemophilia Genotyping Initiative

Jill M Johnsen, MD, Shelley N Fletcher, Angela Dove, Haley McCracken, Beth K Martin, Martin Kircher, Neil C Josephson, MD, Jay Shendure, Sarah Ruuska, MPH, Leonard A. Valentino, MD, Glenn Pierce, MD PhD, Crystal Watson, BS, Dunlei Cheng, PhD, Michael Recht, MD PhD, Barbara A. Konkle, MD

Blood (2020) 136 (Supplement 1): 19.

https://doi.org/10.1182/blood-2020-140649

Published: 2020

Journal Articles

Design of the Von Willebrand Factor in Pregnancy (VIP) Study

Jill M Johnsen, MD, Sarah Ruuska, MPH, Peter A. Kouides, MD, Barbara A. Konkle, MD

Blood (2020) 136 (Supplement 1): 29.

https://doi.org/10.1182/blood-2020-137738

Published: 2020

Journal Articles

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults with Severe Hemophilia a

Andrew D. Leavitt, MD, Barbara A. Konkle, MD, Kimo Stine, MD, Nathan Visweshwar, MD, Thomas J. Harrington, MD, Adam Giermasz, MD PhD, Steven Arkin, MD, Annie Fang, MD PhD, Frank Plonski, RN, MA, Lynne Smith, MBA, Li-Jung Tseng, PhD MBA, Gregory Di Russo, MD, Bettina M Cockroft, MD, Jeremy Rupon, MD, Didier Rouy, MD PhD

Blood (2020) 136 (Supplement 1): 12.

https://doi.org/10.1182/blood-2020-137648

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

Hematology Utilization Group Studies Part VII (HUGS VII): Costs and Impact of Disease in Older Persons with Hemophilia

Randall Curtis, MBA, Marilyn J Manco-Johnson, MD, Barbara A. Konkle, MD, Roshni Kulkarni, MD, Joanne Wu, MDMS, Judith Baker, DrPH, MHSA, Megan M. Ullman, MA, MPH, Duc Quang Tran, Jr., MD, Michael B. Nichol, PhD

Blood (2020) 136 (Supplement 1): 35–36.

https://doi.org/10.1182/blood-2020-141453

Published: 2020

NARROW

Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study

Potential Predictive Biomarkers for the Development and Outcomes of Inhibitors in Hemophilia A Patients

Adherence to Clotting Factor Treatment and Association with Bleeding and Health-Related Quality of Life in Persons with Hemophilia

Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α

Incorporation of O-Glycopeptides into FVIII Decreases Its Immunogenicity

How I approach bleeding in hospitalized patients

Long-Read Sequencing Resolves Complex Structural Variants and Identifies Missing Disease-Causing Variants in Unsolved Cases of Hemophilia

The burden of heavy menstrual bleeding

Depression and Anxiety in Persons with Von Willebrand Disease

Efanesoctocog Alfa Half-Life and Clearance Are Independent of von Willebrand Factor in Severe Hemophilia A: A Post Hoc Analysis from Phase 1/2a Studies

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia a

Bleeding Data across Baseline FIX Expression Levels in People with Hemophilia B: An Analysis Using the 'Factor Expression Study'

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

A Multi-Omics Approach to X-Chromosome Inactivation (XCI) Identifies Severe Xci Skewing in Female Genetic Carriers of Hemophilia

Healthcare Utilization and Health Related Quality of Life in Persons with Von Willebrand Disease

Results of Genetic Analysis of 11,341 Participants Enrolled in the My Life, Our Future (MLOF) Hemophilia Genotyping Initiative

Design of the Von Willebrand Factor in Pregnancy (VIP) Study

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults with Severe Hemophilia a

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

Hematology Utilization Group Studies Part VII (HUGS VII): Costs and Impact of Disease in Older Persons with Hemophilia

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American Society of Hematology

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American Society of Hematology

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