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Journal Articles

Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management in people with hemophilia A or B

Steven W. Pipe, M.D., Toshko Jelev Lissitchkov, Pencho Georgiev, Sarah Mangles, Inga Hegemann, Alice Trinchero, Pratima Chowdary, Adam Forbes, Liqi Feng, Laurel A. Menapace, Salim Kichou, Shauna Rita Andersson, Marek Demissie, Margaret V Ragni

Blood Adv bloodadvances.2024013900.

https://doi.org/10.1182/bloodadvances.2024013900

Published: 2024

Includes: Supplemental data

Journal Articles

Predictors of liver disease outcomes in individuals with hemophilia and HCV infection

Ilias Christodoulou, Scott Rothenberger, Margaret V. Ragni

Blood Adv (2024) 8 (22): 5767–5772.

https://doi.org/10.1182/bloodadvances.2024014350

Published: 2024

Journal Articles

Optimizing liver health before and after gene therapy for hemophilia A

Margaret V. Ragni, Henry Mead, Ype P. de Jong, Radoslaw Kaczmarek, Andrew D. Leavitt, Brian Long, Diane J. Nugent, Denise E. Sabatino, Sylvia Fong, Annette von Drygalski, Christopher E. Walsh, Bruce A. Luxon

Blood Adv (2024) 8 (19): 5203–5212.

https://doi.org/10.1182/bloodadvances.2024013059

Published: 2024

Journal Articles

Long-term clinical outcomes of prophylaxis with an rFVIIIFc or rFIXFc in adults aged ≥50 years with hemophilia A or B

Doris Quon, Shannon Jackson, María Teresa Alvarez-Román, Umer Khan, Sandra Casiano, Margaret V. Ragni, Savita Rangarajan

Blood Adv (2024) 8 (18): 4751–4755.

https://doi.org/10.1182/bloodadvances.2023012462

Published: 2024

Includes: Supplemental data

Journal Articles

Prevalence and risk factors for bleeding in hereditary hemorrhagic telangiectasia: a National Inpatient Sample study

Jabra Zarka, Kwonho Jeong, Jonathan G. Yabes, Margaret V. Ragni

Blood Adv (2023) 7 (19): 5843–5850.

https://doi.org/10.1182/bloodadvances.2023010743

Published: 2023

Journal Articles

Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B

Amy D. Shapiro, Roshni Kulkarni, Margaret V. Ragni, Hervé Chambost, Johnny Mahlangu, Johannes Oldenburg, Beatrice Nolan, Margareth C. Ozelo, Meredith C. Foster, Annemieke Willemze, Christopher Barnowski, Nisha Jain, Bent Winding, Jennifer Dumont, Stefan Lethagen, Chris Barnes, K. John Pasi

Blood Adv (2023) 7 (13): 3049–3057.

https://doi.org/10.1182/bloodadvances.2022009230

Published: 2023

Includes: Supplemental data

Journal Articles

Valoctocogene roxaparvovec gene transfer in participants with HIV

Margaret V. Ragni, Elaine Majerus, Sylvia Fong, Bridget Yates, Stephen Scheeler, Lisa Razon, Hua Yu, Divya B. Reddy, Tara M. Robinson

Blood Adv (2023) 7 (8): 1525–1530.

https://doi.org/10.1182/bloodadvances.2022008948

Published: 2023

Includes: Supplemental data

Journal Articles

Defenestrated endothelium delays liver-directed gene transfer in hemophilia A mice

Tomasz W. Kaminski, Eun-Mi Ju, Shweta Gudapati, Ravi Vats, Sanya Arshad, Rikesh K. Dubey, Omika Katoch, Egemen Tutuncuoglu, Jonathan Frank, Tomasz Brzoska, Donna B. Stolz, Simon C. Watkins, Stephen Y. Chan, Margaret V. Ragni, Enrico M. Novelli, Prithu Sundd, Tirthadipa Pradhan-Sundd

Blood Adv (2022) 6 (12): 3729–3734.

https://doi.org/10.1182/bloodadvances.2021006388

Published: 2022

Includes: Multimedia, Supplemental data

Journal Articles

Whole-exome analysis of adolescents with low VWF and heavy menstrual bleeding identifies novel genetic associations

Brooke Sadler, Charles G. Minard, Gabe Haller, Christina A. Gurnett, Sarah H. O’Brien, Allison Wheeler, Shilpa Jain, Mutka Sharma, Ayesha Zia, Roshni Kulkarni, Eric Mullins, Margaret V. Ragni, Robert Sidonio, Jennifer E. Dietrich, Peter A. Kouides, Jorge Di Paola, Lakshmi Srivaths

Blood Adv (2022) 6 (2): 420–428.

https://doi.org/10.1182/bloodadvances.2021005118

Published: 2022

Includes: Supplemental data

Journal Articles

The design of a Bayesian platform trial to prevent and eradicate inhibitors in patients with hemophilia

Marnie Bertolet, Maria M. Brooks, Margaret V. Ragni

Blood Adv (2020) 4 (21): 5433–5441.

https://doi.org/10.1182/bloodadvances.2020002789

Published: 2020

Includes: Supplemental data

Journal Articles

Recombinant vs plasma-derived von Willebrand factor to prevent postpartum hemorrhage in von Willebrand disease

Nicoletta Machin, Margaret V. Ragni

Blood Adv (2020) 4 (14): 3234–3238.

https://doi.org/10.1182/bloodadvances.2020002046

Published: 2020

Journal Articles

The spectrum and severity of bleeding in adolescents with low von Willebrand factor–associated heavy menstrual bleeding

Lakshmi Srivaths, Charles G. Minard, Sarah H. O’Brien, Allison P. Wheeler, Eric Mullins, Mukta Sharma, Robert Sidonio, Shilpa Jain, Ayesha Zia, Margaret V. Ragni, Roshni Kulkarni, Jennifer E. Dietrich, Peter A. Kouides

Blood Adv (2020) 4 (13): 3209–3216.

https://doi.org/10.1182/bloodadvances.2020002081

Published: 2020

Journal Articles

Emicizumab use in major orthopedic surgery

Craig D. Seaman, Margaret V. Ragni

Blood Adv (2019) 3 (11): 1722–1724.

https://doi.org/10.1182/bloodadvances.2019000228

Published: 2019

Journal Articles

Gene therapy in hemophilia A: a cost-effectiveness analysis

Nicoletta Machin, Margaret V. Ragni, Kenneth J. Smith

Blood Adv (2018) 2 (14): 1792–1798.

https://doi.org/10.1182/bloodadvances.2018021345

Published: 2018

Journal Articles

Sequential bypassing agents during major orthopedic surgery: a new approach to hemostasis

Craig D. Seaman, Margaret V. Ragni

Blood Adv (2017) 1 (17): 1309–1311.

https://doi.org/10.1182/bloodadvances.2017008409

Published: 2017

Journal Articles

Blood volume–based von Willebrand factor to prevent postpartum hemorrhage in von Willebrand disease

Margaret V. Ragni

Blood Adv (2017) 1 (11): 703–706.

https://doi.org/10.1182/bloodadvances.2017005090

Published: 2017

Journal Articles

Accurate, simple, and inexpensive assays to diagnose F8 gene inversion mutations in hemophilia A patients and carriers

Debargh Dutta, Devi Gunasekera, Margaret V. Ragni, Kathleen P. Pratt

Blood Adv (2016) 1 (3): 231–239.

https://doi.org/10.1182/bloodadvances.2016001651

Published: 2016

NARROW

Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management in people with hemophilia A or B

Predictors of liver disease outcomes in individuals with hemophilia and HCV infection

Optimizing liver health before and after gene therapy for hemophilia A

Long-term clinical outcomes of prophylaxis with an rFVIIIFc or rFIXFc in adults aged ≥50 years with hemophilia A or B

Prevalence and risk factors for bleeding in hereditary hemorrhagic telangiectasia: a National Inpatient Sample study

Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B

Valoctocogene roxaparvovec gene transfer in participants with HIV

Defenestrated endothelium delays liver-directed gene transfer in hemophilia A mice

Whole-exome analysis of adolescents with low VWF and heavy menstrual bleeding identifies novel genetic associations

The design of a Bayesian platform trial to prevent and eradicate inhibitors in patients with hemophilia

Recombinant vs plasma-derived von Willebrand factor to prevent postpartum hemorrhage in von Willebrand disease

The spectrum and severity of bleeding in adolescents with low von Willebrand factor–associated heavy menstrual bleeding

Emicizumab use in major orthopedic surgery

Gene therapy in hemophilia A: a cost-effectiveness analysis

Sequential bypassing agents during major orthopedic surgery: a new approach to hemostasis

Blood volume–based von Willebrand factor to prevent postpartum hemorrhage in von Willebrand disease

Accurate, simple, and inexpensive assays to diagnose F8 gene inversion mutations in hemophilia A patients and carriers

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American Society of Hematology

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ASH Publications

American Society of Hematology

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