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1.

Innovations in RNA therapy for hemophilia.

Ragni, Margaret V; Chan, Stephen Y.

Blood; 142(19): 1613-1621, 2023 11 09.

Artículo en Inglés | MEDLINE | ID: mdl-37478403

2.

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.

George, Lindsey A; Monahan, Paul E; Eyster, M Elaine; Sullivan, Spencer K; Ragni, Margaret V; Croteau, Stacy E; Rasko, John E J; Recht, Michael; Samelson-Jones, Benjamin J; MacDougall, Amy; Jaworski, Kristen; Noble, Robert; Curran, Marla; Kuranda, Klaudia; Mingozzi, Federico; Chang, Tiffany; Reape, Kathleen Z; Anguela, Xavier M; High, Katherine A.

N Engl J Med; 385(21): 1961-1973, 2021 11 18.

Artículo en Inglés | MEDLINE | ID: mdl-34788507

3.

Observational cohort study of long-term outcomes of liver transplantation in haemophilia.

Ragni, Margaret V; Callis, Jessica; Daoud, Nabil; Hu, Brian; Manuel, Matthew; Santos, Jarren; Schwartz, Jonathan; Friedman, Kenneth D; Kouides, Peter; Kuriakose, Philip; Leavitt, Andrew D; Lim, Ming Y; Machin, Nicoletta; Recht, Michael; Chrisentery-Singleton, Tammuella.

Haemophilia; 30(1): 87-97, 2024 Jan.

Artículo en Inglés | MEDLINE | ID: mdl-38111071

4.

BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A.

Konkle, Barbara A; Shapiro, Amy D; Quon, Doris V; Staber, Janice M; Kulkarni, Roshni; Ragni, Margaret V; Chhabra, Ekta S; Poloskey, Stacey; Rice, Kara; Katragadda, Suresh; Fruebis, Joachim; Benson, Craig C.

N Engl J Med; 383(11): 1018-1027, 2020 09 10.

Artículo en Inglés | MEDLINE | ID: mdl-32905674

5.

Prevalence and risk factors for hepatocellular carcinoma in individuals with haemophilia in the era of direct-acting antiviral agents: A national inpatient sample study.

Yang, Xi; Jeong, Kwonho; Yabes, Jonathan G; Ragni, Margaret V.

Haemophilia; 28(5): 769-775, 2022 Sep.

Artículo en Inglés | MEDLINE | ID: mdl-35727998

6.

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

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.

Haemophilia; 28(3): 453-461, 2022 May.

Artículo en Inglés | MEDLINE | ID: mdl-35263495

7.

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 crossover trial.

Seaman, Craig D; Yabes, Jonathan G; Lalama, Christina M; Ragni, Margaret V.

Haemophilia; 27(3): 351-357, 2021 May.

Artículo en Inglés | MEDLINE | ID: mdl-33749970

8.

Quality of periprocedural care in patients with von Willebrand disease.

Seaman, Craig D; Bertolet, Marnie; Zhang, Jun; Ragni, Margaret V.

Haemophilia; 27(5): 830-836, 2021 Sep.

Artículo en Inglés | MEDLINE | ID: mdl-34343372

9.

Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural).

Shapiro, Amy D; Ragni, Margaret V; Borhany, Munira; Abajas, Yasmina L; Tarantino, Michael D; Holstein, Katharina; Croteau, Stacy E; Liesner, Riana; 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; Donfield, Sharyne; LeBeau, Petra; Berntorp, Erik; Astermark, Jan.

Haemophilia; 27(1): 49-59, 2021 Jan.

Artículo en Inglés | MEDLINE | ID: mdl-33278853

10.

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.

George, Lindsey A; Ragni, Margaret V; Rasko, John E J; Raffini, Leslie J; Samelson-Jones, Benjamin J; Ozelo, Margareth; Hazbon, Maria; Runowski, Alexa R; Wellman, Jennifer A; Wachtel, Katie; Chen, Yifeng; Anguela, Xavier M; Kuranda, Klaudia; Mingozzi, Federico; High, Katherine A.

Mol Ther; 28(9): 2073-2082, 2020 09 02.

Artículo en Inglés | MEDLINE | ID: mdl-32559433

11.

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.

Pasi, K John; Rangarajan, Savita; Georgiev, Pencho; Mant, Tim; Creagh, Michael D; Lissitchkov, Toshko; Bevan, David; Austin, Steve; Hay, Charles R; Hegemann, Inga; Kazmi, Rashid; Chowdary, Pratima; Gercheva-Kyuchukova, Liana; Mamonov, Vasily; Timofeeva, Margarita; Soh, Chang-Heok; Garg, Pushkal; Vaishnaw, Akshay; Akinc, Akin; Sørensen, Benny; Ragni, Margaret V.

N Engl J Med; 377(9): 819-828, 2017 08 31.

Artículo en Inglés | MEDLINE | ID: mdl-28691885

12.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

George, Lindsey A; Sullivan, Spencer K; Giermasz, Adam; Rasko, John E J; Samelson-Jones, Benjamin J; Ducore, Jonathan; Cuker, Adam; Sullivan, Lisa M; Majumdar, Suvankar; Teitel, Jerome; McGuinn, Catherine E; Ragni, Margaret V; Luk, Alvin Y; Hui, Daniel; Wright, J Fraser; Chen, Yifeng; Liu, Yun; Wachtel, Katie; Winters, Angela; Tiefenbacher, Stefan; Arruda, Valder R; van der Loo, Johannes C M; Zelenaia, Olga; Takefman, Daniel; Carr, Marcus E; Couto, Linda B; Anguela, Xavier M; High, Katherine A.

N Engl J Med; 377(23): 2215-2227, 2017 12 07.

Artículo en Inglés | MEDLINE | ID: mdl-29211678

13.

Traumatic injury results in prolonged circulation of ultralarge von Willebrand factor and a reduction in ADAMTS13 activity.

Dyer, Mitchell R; Plautz, William E; Ragni, Margaret V; Alexander, Wyeth; Haldeman, Shannon; Sperry, Jason L; Guyette, Francis X; Zuckerbraun, Brian S; Rollins-Raval, Marian A; Raval, Jay S; Neal, Matthew D.

Transfusion; 60(6): 1308-1318, 2020 06.

Artículo en Inglés | MEDLINE | ID: mdl-32441353

14.

Emicizumab prophylaxis in patients with haemophilia A with and without inhibitors.

Ebbert, Patrick T; Xavier, Frederico; Seaman, Craig D; Ragni, Margaret V.

Haemophilia; 26(1): 41-46, 2020 Jan.

Artículo en Inglés | MEDLINE | ID: mdl-31746522

15.

The impact of extended half-life factor concentrates on prophylaxis for severe hemophilia in the United States.

Malec, Lynn M; Cheng, Dunlei; Witmer, Char M; Jaffray, Julie; Kouides, Peter A; Haley, Kristina M; Sidonio, Robert F; Johnson, Kelsey; Recht, Michael; White, Gilbert; Croteau, Stacy E; Ragni, Margaret V.

Am J Hematol; 95(8): 960-965, 2020 08.

Artículo en Inglés | MEDLINE | ID: mdl-32356313

16.

The national blueprint for future factor VIII inhibitor clinical trials: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors.

Ragni, Margaret V; George, Lindsey A.

Haemophilia; 25(4): 581-589, 2019 Jul.

Artículo en Inglés | MEDLINE | ID: mdl-31329364

17.

Incidence and risk factors for hepatocellular cancer in individuals with haemophilia: A National Inpatient Sample Study.

Thalappillil, Alvin; Ragni, Margaret V; Comer, Diane M; Yabes, Jonathan G.

Haemophilia; 25(2): 221-228, 2019 Mar.

Artículo en Inglés | MEDLINE | ID: mdl-30615816

18.

Platelet VIII pack evades immune detection.

Ragni, Margaret V.

Blood; 127(10): 1222-4, 2016 Mar 10.

Artículo en Inglés | MEDLINE | ID: mdl-26965921

19.

Clinical and laboratory variability in a cohort of patients diagnosed with type 1 VWD in the United States.

Flood, Veronica H; Christopherson, Pamela A; Gill, Joan Cox; Friedman, Kenneth D; Haberichter, Sandra L; Bellissimo, Daniel B; Udani, Rupa A; Dasgupta, Mahua; Hoffmann, Raymond G; Ragni, Margaret V; Shapiro, Amy D; Lusher, Jeanne M; Lentz, Steven R; Abshire, Thomas C; Leissinger, Cindy; Hoots, W Keith; Manco-Johnson, Marilyn J; Gruppo, Ralph A; Boggio, Lisa N; Montgomery, Kate T; Goodeve, Anne C; James, Paula D; Lillicrap, David; Peake, Ian R; Montgomery, Robert R.

Blood; 127(20): 2481-8, 2016 05 19.

Artículo en Inglés | MEDLINE | ID: mdl-26862110

20.

Prophylactic rtPA in the Prevention of Line-associated Thrombosis and Infection in Short Bowel Syndrome.

Malec, Lynn M; Cooper, James; Rudolph, Jeffrey; Michaels, Marian G; Ragni, Margaret V.

J Pediatr Gastroenterol Nutr; 66(6): 972-975, 2018 Jun.

Artículo en Inglés | MEDLINE | ID: mdl-29135819

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