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Journal Articles

Royal gene therapy at a royal cost

Margaret Ragni

Blood (2021) 138 (18): 1645–1646.

https://doi.org/10.1182/blood.2021012129

Published: 2021

Journal Articles

Gene-produced FVIII: measure for measure

Margaret Ragni

Blood (2020) 136 (22): 2483–2484.

https://doi.org/10.1182/blood.2020008974

Published: 2020

Journal Articles

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

Margaret Ragni, Roshni Kulkarni, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Kathelijn Fischer, Johnny Mahlangu, Amy Shapiro, Beatrice Nolan, Johannes Oldenburg, Tadashi Matsushita, Annemieke Willemze, Huixing Yuan, Dan Rudin

Blood (2018) 132 (Supplement 1): 1214.

https://doi.org/10.1182/blood-2018-99-110100

Published: 2018

Journal Articles

Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease

Joan C. Gill, Giancarlo Castaman, Jerzy Windyga, Peter Kouides, Margaret Ragni, Frank W. G. Leebeek, Ortrun Obermann-Slupetzky, Miranda Chapman, Sandor Fritsch, Borislava G. Pavlova, Isabella Presch, Bruce Ewenstein

Blood (2015) 126 (17): 2038–2046.

https://doi.org/10.1182/blood-2015-02-629873

Published: 2015

Journal Articles

Pharmacokinetic Evaluation of Von Willebrand Factor/Factor VIII Concentrate (Humate-P®) Prior to Elective Surgery in Patients with Von Willebrand Disease.

Jorge Di Paola, MD, Joan Gill, MD, Jonathan Bernstein, MD, Cindy Leissinger, MD, Margaret Ragni, MD, Rinah Shopnik, MD, Leonard Valentino, MD, Carl Friedman, MD, Sigurd Knaub, MD

Blood (2006) 108 (11): 4077.

https://doi.org/10.1182/blood.V108.11.4077.4077

Published: 2006

Journal Articles

Predictors of Liver Fibrosis in HCV+ Hemophilic Men.

Kakra Soadwa, MPH, Michael Nalesnik, MD, Qianyu Dang, PhD, Margaret Ragni, MD

Blood (2005) 106 (11): 319.

https://doi.org/10.1182/blood.V106.11.319.319

Published: 2005

Journal Articles

Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B.

Katherine High, MD, Michael Tigges, PhD, Catherine Manno, MD, Denise Sabatino, PhD, Valder Arruda, MD, Roland Herzog, PhD, Pradip Rustagi, MD, John Rasko, MD, Jurg Sommer, PhD, Kristen Jaworski, RN, Margaret Ragni, MD, Bertil Glader, MD, Ruth Lessard, Alvin Luk, PhD, Linda Couto, PhD, Haiyan Jiang, PhD, Glenn Pierce, MD, Mark Kay, MD

Blood (2004) 104 (11): 413.

https://doi.org/10.1182/blood.V104.11.413.413

Published: 2004

NARROW

Royal gene therapy at a royal cost

Gene-produced FVIII: measure for measure

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease

Pharmacokinetic Evaluation of Von Willebrand Factor/Factor VIII Concentrate (Humate-P®) Prior to Elective Surgery in Patients with Von Willebrand Disease.

Predictors of Liver Fibrosis in HCV+ Hemophilic Men.

Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B.

Email alerts

ASH Publications

American Society of Hematology

Royal gene therapy at a royal cost

Gene-produced FVIII: measure for measure

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease

Pharmacokinetic Evaluation of Von Willebrand Factor/Factor VIII Concentrate (Humate-P®) Prior to Elective Surgery in Patients with Von Willebrand Disease.

Predictors of Liver Fibrosis in HCV+ Hemophilic Men.

Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B.

Email alerts

ASH Publications

American Society of Hematology

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