MargaretVRagniMD

PubMed

• Observational cohort study of long-term outcomes of liver transplantation in haemophilia.
  Callis, J., Daoud, N., Hu, B., Manuel, M., Santos, J., Schwartz, J., Friedman, K., Kuriakose, P., Leavitt, A., Lim, M., Recht, M., Ragni, M., Kouides, P., Machin, N., ...> ;Haemophilia. 2024 Jan 1
• Rapidly loading emicizumab without immunosuppression in acquired haemophilia.
  Margaret V Ragni> ;The Lancet. Haematology. 2023 Nov 1
• 1 citations
  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 Advances. 2023 Oct 10
• Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults.
  Barbara A Konkle, Johannes Oldenburg, John Pasi, Roshni Kulkarni, Beatrice Nolan, Johnny Mahlangu, Guy Young, Simon A Brown, Ingrid Pabinger, Amy Shapiro, Claude Négri...> ;Research and Practice in Thrombosis and Haemostasis. 2023 Aug 1
• Long-term outcomes of prophylaxis with a recombinant factor VIII Fc or recombinant factor IX Fc in patients with hemophilia previously treated on demand.
  María-Teresa Álvarez-Román, Amy D Shapiro, Margaret V Ragni, Helena Palmborg, Linda Bystrická, Johan Szamosi, Sandra Casiano, Hervé Chambost> ;Research and Practice in Thrombosis and Haemostasis. 2023 Aug 1
• 1 citations
  Innovations in RNA therapy for hemophilia.
  Margaret V Ragni, Stephen Y Chan> ;Blood. 2023 Nov 9
• 6 citations
  Recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in patients with mild and moderate von Willebrand disease in the USA (VWDMin): a pha...
  Margaret V Ragni, Scott D Rothenberger, Robert Feldman, Danielle Nance, Andrew D Leavitt, Lynn Malec, Roshni Kulkarni, Robert Sidonio Jr, Eric Kraut, Joseph Lasky, Raj...> ;The Lancet. Haematology. 2023 Aug 1
• 5 citations
  A review of the rationale for gene therapy for hemophilia A with inhibitors: one-shot tolerance and treatment?
  Leonard A Valentino, Margareth C Ozelo, Roland W Herzog, Nigel S Key, Allyson M Pishko, Margaret V Ragni, Benjamin J Samelson-Jones, David Lillicrap> ;Journal of Thrombosis and Haemostasis. 2023 Nov 1
• 8 citations
  Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce...
  Margaret V Ragni, Guy Young, Glaivy Batsuli, Emily Bisson, Shannon L Carpenter, Stacy E Croteau, Adam Cuker, Randall G Curtis, Michael Denne, Bruce Ewenstein, Amber Fe...> ;Expert Review of Hematology. 2023 Mar 1
• 1 citations
  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 ...> ;Blood Advances. 2023 Jul 11
• 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 Advances. 2023 Apr 25
• 1 citations
  Hemophilia A subjects with an intron-22 gene inversion mutation show CD4T-effector responses to multiple epitopes in FVIII.
  Devi Gunasekera, Pooja Vir, Ahmad Faisal Karim, Margaret V Ragni, Kathleen P Pratt> ;Frontiers in Immunology. 2023 Jan 1
• 3 citations
  Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies.
  Pratima Chowdary, Margareta Holmström, Johnny N Mahlangu, Margaret C Ozelo, Ingrid Pabinger, K John Pasi, Margaret V Ragni, Amy Shapiro, Chris Barnowski, Stefan Lethagen> ;Research and Practice in Thrombosis and Haemostasis. 2022 Jul 1
• 4 citations
  Prevalence and risk factors for hepatocellular carcinoma in individuals with haemophilia in the era of direct-acting antiviral agents: A national inpatient sample study.
  Xi Yang, Kwonho Jeong, Jonathan G Yabes, Margaret V Ragni> ;Haemophilia. 2022 Sep 1
• 3 citations
  Quality of life in a large multinational haemophilia B cohort (The B-Natural study) - Unmet needs remain.
  Erik Berntorp, Petra LeBeau, Margaret V Ragni, Munira Borhany, Yasmina L Abajas, Michael D Tarantino, Katharina Holstein, Stacy E Croteau, Raina Liesner, Cristina Tara...> ;Haemophilia. 2022 May 1
• 2 citations
  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...> ;Blood Advances. 2022 Jun 28
• 14 citations
  Postoperative bleeding complications in patients with hemophilia undergoing major orthopedic surgery: A prospective multicenter observational study.
  Brendan Kleiboer, Marcus A Layer, Lorraine A Cafuir, Adam Cuker, Miguel Escobar, M Elaine Eyster, Eric Kraut, Andrew D Leavitt, Steven R Lentz, Doris Quon, Margaret V ...> ;Journal of Thrombosis and Haemostasis. 2022 Apr 1
• 3 citations
  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 P. Wheeler, Shilpa Jain, Mukta Sharma, Ayesha Zia, Roshni Kulkarni, Eric...> ;Blood Advances. 2021 Nov 22
• 1 citations
  Royal gene therapy at a royal cost.
  Margaret V. Ragni> ;Blood. 2021 Nov 4
• 9 citations
  Hemophilia as a blueprint for gene therapy.
  Margaret V. Ragni> ;Science. 2021 Sep 30
• 3 citations
  Hemophilia A (Factor VIII Deficiency).
  Craig D. Seaman, Frederico Xavier, Margaret V. Ragni> ;Hematology/oncology Clinics of North America. 2021 Aug 10
• 3 citations
  Quality of periprocedural care in patients with von Willebrand disease.
  Craig D. Seaman, Marnie Bertolet, Jun Zhang, Margaret V. Ragni> ;Haemophilia. 2021 Aug 3
• 14 citations
  Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B
  Beatrice Nolan, Anna Klukowska, Amy D. Shapiro, Antoine Rauch, Michael Recht, Margaret V. Ragni, Julie Curtin, Sriya Gunawardena, Sutirtha Mukhopadhyay, Deepthi Jayawa...> ;Blood Advances. 2021 Jul 13
• 1 citations
  Optical or optimal conclusion: pharmacokinetic-guided dosing in haemophilia
  Margaret V. Ragni> ;The Lancet. Haematology. 2021 Jul 1
• 1 citations
  Factor VIII concentrate dosing with lean body mass, ideal body weight and total body weight in overweight and obesity: A randomized, controlled, open-label, 3 × 3 cros...
  Craig D. Seaman, Jonathan G. Yabes, Christina M. Lalama, Margaret V. Ragni> ;Haemophilia. 2021 May 1
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Journal Articles

• BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A  
  Doris V Quon, Margaret V Ragni, The New England Journal of Medicine

Abstracts/Posters

• Innate Immune Mechanism of Hemarthrosis in Hemophilia-a Mice
  Margaret V. Ragni, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
• Mechanism of Pulmonary Thrombosis in Hemolytic Disorders
  Margaret V. Ragni, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
• FVIII Protein Is Not Detectable in Human PBMCs or Livers from Dogs with an Intron-22 Inversion Mutation: Implications for FVIII Immunogenicity and Tolerance
  Margaret V. Ragni, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
• Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute ...
  Margaret V. Ragni, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
• Emicizumab Prophylaxis in Patients with Hemophilia _ with and without Inhibitors
  Margaret V. Ragni, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/8/2019
• Prolonged Circulation of Ultra-Large Von Willebrand Factor and a Reduction in ADAMTS13 Activity Promotes Microvascular Disease Following Traumatic Injury
  Margaret V. Ragni, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
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Lectures

• Hemostasis and Thrombosis 
  61st Annual American Society of Hematology Meeting - Orlando, FL - 12/7/2019
• Von Willebrand Disease Minimize Menorrhagia (VWDMin) Trial 
  61st Annual American Society of Hematology Meeting - Orlando, FL - 12/7/2019
• Coagulation Conundrum Roundtable: Case-based Discussion on the Implications of Exogenous Estrogens in Hemostasis and Thrombosis 
  61st Annual American Society of Hematology Meeting - Orlando, FL - 12/7/2019
• Real World Use of Extended Half-Life Products and the Impact on Bleeding Events and Joint Health in the United States 
  2018 ASH Annual Meeting - San Diego, CA - 12/1/2018
• Von Willebrand Disease and Gastrointestinal Bleeding: A National Inpatient Sample (NIS) Study 
  2018 ASH Annual Meeting - San Diego, CA - 12/1/2018
• Scientific Communication: Explaining Your Science to a Lay Audience 
  60th American Society of Hematology Annual Meeting - 11/30/2018
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