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Dr. Ragni has actively pursued, initiated, collaborated, and participated in clinical translational research in congenital hemostasis and thrombosis disorders, serving as chair of clinical trials, prospective epidemiologic, observational, case-control studies, cost-effectiveness analyses, and investigator-initiated new drug trials in hemophilia and VWD. Her research studies were among the first multi-center NIH-funded investigator-initiated studies in hemophilia malignancy (NCI), hemophilia inhibitor formation (NHLBI), hemophilia HIV/HCV infection (NHLBI), hemophilia AIDS therapy (NIAID), and hemophilia adult prophylaxis (NHLBI). She co-chaired the NHLBI State of the Science (SOS) Inhibitor Prevention Trial Working Group (2018), and serves as PI of the Inhibitor Prevention and Eradication Platform Trial (HRSA). She also co-chaired the NHLBI SOS Hemophilia & VWD Subcommittee to design future trials, with three U34 trials funded NHLBI, and the VWDMin NHLBI U01 clinical trial to reduce bleeding in women with VWD, and the VWD-Woman trial prevent VWD postpartum bleeding (Takeda). She has collaborated on multi-center organ transplant HIV trials (NIAID), hemophilia gene therapy trials (NHLBI); VWD genotype-phenotype studies (NHLBI); novel therapeutics (fitusiran (siRNA AT) and hemlibra (emcizumab) and extended half-life protein trials (VIIa, VIII, IX) for hemophilia, and rhIL-11 and recombinant VWF for VWD. She has 9 active NIH grants, two of which she serves as PI; and local PI on 6 ATHN CDC grants.
研究兴趣
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Margaret V. Ragni, Jessica Callis,Nabil Daoud,Brian Hu, Matthew Manuel, Jarren Santos, Jonathan Schwartz, Kenneth D. Friedman,Peter Kouides,Philip Kuriakose,Andrew D. Leavitt,Ming Y. Lim,
The Lancet. Haematologyno. 11 (2023): E870-E871
JOURNAL OF THROMBOSIS AND HAEMOSTASISno. 11 (2023): 3033-3044
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 Federizo,Neil Frick,
Expert review of hematologyno. sup1 (2023): 107-127
L. Skeith,P. James,P. Kouides,K. Uminski,L. Duffett,S. Jackson,M. Sholzberg,M. Ragni,A. Cuker, M. O’Beirne, S. Rubinstein,J. Hews-Girard,
Research and Practice in Thrombosis and Haemostasis (2023): 100673-100673
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