An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B

por Machin, Nicoletta, Ragni, Margaret V
Publicado 2018

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

por Zarka, Jabra, Jeong, Kwonho, Yabes, Jonathan G., Ragni, Margaret V.
Publicado 2023

Unfractionated heparin in acute chest syndrome: a pilot feasibility randomized controlled trial of unfractionated heparin vs. standard of care in acute chest syndrome

por Seaman, Craig D., Novelli, Enrico, De Castro, Laura, Ragni, Margaret V.
Publicado 2020

Exploring the Complex Role of Coagulation Factor VIII in Chronic Liver Disease

por Pradhan-Sundd, Tirthadipa, Gudapati, Shweta, Kaminski, Tomasz W., Ragni, Margaret V.
Publicado 2021

Inflammatory Bowel Disease and Thrombosis: A National Inpatient Sample Study

por Cohen, Jessica B., Comer, Diane M., Yabes, Jonathan G., Ragni, Margaret V.
Publicado 2020

Hemophilia A subjects with an intron-22 gene inversion mutation show CD4(+) T-effector responses to multiple epitopes in FVIII

por Gunasekera, Devi, Vir, Pooja, Karim, Ahmad Faisal, Ragni, Margaret V., Pratt, Kathleen P.
Publicado 2023

Long-term outcomes of prophylaxis with a recombinant factor VIII Fc or recombinant factor IX Fc in patients with hemophilia previously treated on demand

por Álvarez-Román, María-Teresa, Shapiro, Amy D., Ragni, Margaret V., Palmborg, Helena, Bystrická, Linda, Szamosi, Johan, Casiano, Sandra, Chambost, Hervé
Publicado 2023

Valoctocogene roxaparvovec gene transfer in participants with HIV

por Ragni, Margaret V., Majerus, Elaine, Fong, Sylvia, Yates, Bridget, Scheeler, Stephen, Razon, Lisa, Yu, Hua, Reddy, Divya B., Robinson, Tara M.
Publicado 2023

Porcine and Canine von Willebrand Factor and von Willebrand Disease: Hemostasis, Thrombosis, and Atherosclerosis Studies

por Nichols, Timothy C., Bellinger, Dwight A., Merricks, Elizabeth P., Raymer, Robin A., Kloos, Mark T., DeFriess, Natalie, Ragni, Margaret V., Griggs, Thomas R.
Publicado 2010

Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies

por Chowdary, Pratima, Holmström, Margareta, Mahlangu, Johnny N., Ozelo, Margaret C., Pabinger, Ingrid, Pasi, K. John, Ragni, Margaret V., Shapiro, Amy, Barnowski, Chris, Lethagen, Stefan
Publicado 2022

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

por Konkle, Barbara A., Oldenburg, Johannes, Pasi, John, Kulkarni, Roshni, Nolan, Beatrice, Mahlangu, Johnny, Young, Guy, Brown, Simon A., Pabinger, Ingrid, Shapiro, Amy, Négrier, Claude, Blanchette, Victor, Ragni, Margaret V., Dumont, Jennifer, Lethagen, Stefan
Publicado 2023

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort

por Pasi, K. John, Lissitchkov, Toshko, Mamonov, Vasily, Mant, Tim, Timofeeva, Margarita, Bagot, Catherine, Chowdary, Pratima, Georgiev, Pencho, Gercheva‐Kyuchukova, Liana, Madigan, Kate, Van Nguyen, Huy, Yu, Qifeng, Mei, Baisong, Benson, Craig C., Ragni, Margaret V.
Publicado 2021

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

por Shapiro, Amy D., Kulkarni, Roshni, Ragni, Margaret V., Chambost, Hervé, Mahlangu, Johnny, Oldenburg, Johannes, Nolan, Beatrice, Ozelo, Margareth C., Foster, Meredith C., Willemze, Annemieke, Barnowski, Christopher, Jain, Nisha, Winding, Bent, Dumont, Jennifer, Lethagen, Stefan, Barnes, Chris, Pasi, K. John
Publicado 2023

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

por Sadler, Brooke, Minard, Charles G., Haller, Gabe, Gurnett, Christina A., O’Brien, Sarah H., Wheeler, Allison, Jain, Shilpa, Sharma, Mutka, Zia, Ayesha, Kulkarni, Roshni, Mullins, Eric, Ragni, Margaret V., Sidonio, Robert, Dietrich, Jennifer E., Kouides, Peter A., Di Paola, Jorge, Srivaths, Lakshmi
Publicado 2022

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

por Kaminski, Tomasz W., Ju, Eun-Mi, Gudapati, Shweta, Vats, Ravi, Arshad, Sanya, Dubey, Rikesh K., Katoch, Omika, Tutuncuoglu, Egemen, Frank, Jonathan, Brzoska, Tomasz, Stolz, Donna B., Watkins, Simon C., Chan, Stephen Y., Ragni, Margaret V., Novelli, Enrico M., Sundd, Prithu, Pradhan-Sundd, Tirthadipa
Publicado 2022

Quality of life in a large multinational haemophilia B cohort (The B‐Natural study) – Unmet needs remain

por Berntorp, Erik, LeBeau, Petra, Ragni, Margaret V., Borhany, Munira, Abajas, Yasmina L., Tarantino, Michael D., Holstein, Katharina, Croteau, Stacy E., Liesner, Raina, Tarango, Cristina, Carvalho, Manuela, McGuinn, Catherine, Funding, Eva, Kempton, Christine L., Bidlingmaier, Christoph, Cohen, Alice, Oldenburg, Johannes, Kearney, Susan, Knoll, Christine, Kuriakose, Philip, Acharya, Suchitra, Reiss, Ulrike M., Kulkarni, Roshni, Witkop, Michelle, Lethagen, Stefan, Krouse, Rebecca, Shapiro, Amy D., Astermark, Jan
Publicado 2022