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.
Published in Molecular therapy (02.09.2020)
Published in Molecular therapy (02.09.2020)
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Journal Article
Headache in Children with Sickle Cell Disease: Prevalence and Associated Factors
Niebanck, Alison E., MD, Pollock, Avrum N., MD, Smith-Whitley, Kim, MD, Raffini, Leslie J., MD, Zimmerman, Robert A., MD, Ohene-Frempong, Kwaku, MD, Kwiatkowski, Janet L., MD
Published in The Journal of pediatrics (01.07.2007)
Published in The Journal of pediatrics (01.07.2007)
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Journal Article
Venous Thromboembolism in Pediatric Inflammatory Bowel Disease: A Case-Control Study
Mitchel, Elana B, Rosenbaum, Sara, Gaeta, Christopher, Huang, Jing, Raffini, Leslie J, Baldassano, Robert N, Denburg, Michelle R, Albenberg, Lindsey
Published in Journal of pediatric gastroenterology and nutrition (01.05.2021)
Published in Journal of pediatric gastroenterology and nutrition (01.05.2021)
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Efficacy of Early Prophylaxis Against Catheter-Associated Thrombosis in Critically Ill Children: A Bayesian Phase 2b Randomized Clinical Trial
Faustino, E Vincent S, Shabanova, Veronika, Raffini, Leslie J, Kandil, Sarah B, Li, Simon, Pinto, Matthew G, Cholette, Jill M, Hanson, Sheila J, Nellis, Marianne E, Silva, Cicero T, Chima, Ranjit, Sharathkumar, Anjali, Thomas, Kimberly A, McPartland, Tara, Tala, Joana A, Spinella, Philip C
Published in Critical care medicine (01.03.2021)
Published in Critical care medicine (01.03.2021)
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Regional variation and cost implications of prescribed extended half‐life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia
Croteau, Stacy E., Cheng, Dunlei, Cohen, Alice J., Holmes, Chris E., Malec, Lynn M., Silvey, Michael, Thornburg, Courtney D., Wheeler, Allison P., Kouides, Peter A., Raffini, Leslie J., Neufeld, Ellis J.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.07.2019)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.07.2019)
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Age-Dependent Heterogeneity in the Efficacy of Prophylaxis With Enoxaparin Against Catheter-Associated Thrombosis in Critically Ill Children: A Post Hoc Analysis of a Bayesian Phase 2b Randomized Clinical Trial
Faustino, E Vincent S, Raffini, Leslie J, Hanson, Sheila J, Cholette, Jill M, Pinto, Matthew G, Li, Simon, Kandil, Sarah B, Nellis, Marianne E, Shabanova, Veronika, Silva, Cicero T, Tala, Joana A, McPartland, Tara, Spinella, Philip C
Published in Critical care medicine (01.04.2021)
Published in Critical care medicine (01.04.2021)
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Incidence, Management, and Outcomes of Pulmonary Embolism at Tertiary Pediatric Hospitals in the United States
Rastogi, Radhika, Okunowo, Oluwatimilehin, Faerber, Jennifer A., Mavroudis, Constantine D., Whitworth, Hilary, Giglia, Therese M., Witmer, Char, Raffini, Leslie J., O'Byrne, Michael L.
Published in JACC. Advances (Online) (01.04.2024)
Published in JACC. Advances (Online) (01.04.2024)
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Journal Article
Panhandle and Reverse-Panhandle PCR Enable Cloning of Der(11) and Der(other) Genomic Breakpoint Junctions of MLL Translocations and Identify Complex Translocation of MLL, AF-4, and CDK6
Raffini, Leslie J., Slater, Diana J., Rappaport, Eric F., Lo Nigro, Luca, Cheung, Nai-Kong V., Biegel, Jaclyn A., Nowell, Peter C., Lange, Beverly J., Felix, Carolyn A.
Published in Proceedings of the National Academy of Sciences - PNAS (02.04.2002)
Published in Proceedings of the National Academy of Sciences - PNAS (02.04.2002)
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Journal Article
Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant extended half-life FVIII
Kearney, Susan, Raffini, Leslie J, Pham, Tan P, Lee, Xin Ying, von Mackensen, Sylvia, Landorph, Andrea, Takedani, Hideyuki, Oldenburg, Johannes
Published in Patient preference and adherence (01.01.2019)
Published in Patient preference and adherence (01.01.2019)
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Journal Article
Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants
Samelson-Jones, Benjamin J., Finn, Jonathan D., Raffini, Leslie J., Merricks, Elizabeth P., Camire, Rodney M., Nichols, Timothy C., Arruda, Valder R.
Published in Blood advances (09.03.2021)
Published in Blood advances (09.03.2021)
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Journal Article
IVC filter retrieval in adolescents: experience in a tertiary pediatric center
Guzman, Anthony K., Zahra, Mahmoud, Trerotola, Scott O., Raffini, Leslie J., Itkin, Maxim, Keller, Marc S., Cahill, Anne Marie
Published in Pediatric radiology (01.04.2016)
Published in Pediatric radiology (01.04.2016)
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Journal Article
Focusing in on use of pharmacokinetic profiles in routine hemophilia care
Croteau, Stacy E., Callaghan, Michael U., Davis, Joanna, Dunn, Amy L., Guerrera, Michael, Khan, Osman, Neufeld, Ellis J., Raffini, Leslie J., Recht, Michael, Wang, Michael, Iorio, Alfonso
Published in Research and practice in thrombosis and haemostasis (01.07.2018)
Published in Research and practice in thrombosis and haemostasis (01.07.2018)
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Journal Article
Varied opinions on thrombolysis for venous thromboembolism in infants and children: Findings from a survey of pediatric hematology-oncology specialists
Yee, Donald L., Chan, Anthony K.C., Williams, Suzan, Goldenberg, Neil A., Massicotte, M. Patricia, Raffini, Leslie J.
Published in Pediatric blood & cancer (01.12.2009)
Published in Pediatric blood & cancer (01.12.2009)
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