Search Results - "Dunn, Amy L."

An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo by Li, Bin, Luo, Xiao, Deng, Binbin, Wang, Junfeng, McComb, David W, Shi, Yimin, Gaensler, Karin M. L, Tan, Xu, Dunn, Amy L, Kerlin, Bryce A, Dong, Yizhou

“…Systemic delivery of mRNA-based therapeutics remains a challenging issue for preclinical and clinical studies. Here, we describe new lipid-like nanoparticles…”
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Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A by Mahlangu, Johnny, Kaczmarek, Radoslaw, von Drygalski, Annette, Shapiro, Susan, Chou, Sheng-Chieh, Ozelo, Margareth C., Kenet, Gili, Peyvandi, Flora, Wang, Michael, Madan, Bella, Key, Nigel S., Laffan, Michael, Dunn, Amy L., Mason, Jane, Quon, Doris V., Symington, Emily, Leavitt, Andrew D., Oldenburg, Johannes, Chambost, Hervé, Reding, Mark T., Jayaram, Kala, Yu, Hua, Mahajan, Reena, Chavele, Konstantia-Maria, Reddy, Divya B., Henshaw, Joshua, Robinson, Tara M., Wong, Wing Yen, Pipe, Steven W.

“…Participants with hemophilia A received B-domain–deleted factor VIII gene therapy delivered in an AAV5 vector. A decrease in annualized bleeding rates was…”
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Andexanet Alfa Versus Prothrombin Complex Concentrates/Blood Products as Apixaban/Rivaroxaban Reversal Agents: A Survey Among Pediatric Hematologists by Rodriguez, Vilmarie, Stanek, Joseph, Kerlin, Bryce A., Dunn, Amy L.

“…Direct oral factor Xa inhibitors (DXIs) are approved for use in adult patients with non-valvular heart disease for stroke prevention, and the…”
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Efficacy and safety evaluation of eptacog beta (coagulation factor VIIa [recombinant]-jncw) for the treatment of hemophilia A and B with inhibitors by Pipe, Steven W, Dunn, Amy L, Young, Guy

“…Bypassing agents (BPAs) are used to treat acute bleeding episodes, manage bleeding during perioperative care, and prophylactically minimize bleed occurrence in…”
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Venous Thromboembolism in Children with Sickle Cell Disease: A Retrospective Cohort Study by Woods, Gary M., Sharma, Ruchika, Creary, Susan, O'Brien, Sarah, Stanek, Joseph, Hor, Kan, Young, Jennifer, Dunn, Amy L., Kumar, Riten

“…To describe the cumulative incidence of venous thromboembolism (VTE) in children with sickle cell disease (SCD) followed at a single institution and report on…”
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Can virtual reality change the paediatric health-care experience? by Dunn, Amy L

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Mild Hemophilia in a Competitive High School Soccer Player: A Case Report by Omole, Oluwakorede, Reed, Joan, Dunn, Amy L, Gleason, Courtney N

“…In this report, we discuss the case of a now 23-year-old athlete who was diagnosed with mild hemophilia, successfully played varsity soccer throughout high…”
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Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A by Malec, Lynn, Peyvandi, Flora, Chan, Anthony K.C., Königs, Christoph, Zulfikar, Bulent, Yuan, Huixing, Simpson, Mindy, Álvarez Román, Maria Teresa, Carcao, Manuel, Staber, Janice M., Dunn, Amy L., Chou, Sheng-Chieh, d’Oiron, Roseline, Albisetti, Manuela, Demissie, Marek, Santagostino, Elena, Yarramaneni, Abhimanyu, Wong, Nancy, Abad-Franch, Lydia, Gunawardena, Sriya, Fijnvandraat, Karin

“…Efanesoctocog alfa is an engineered form of factor VIII that overcomes the half-life ceiling imposed by von Willebrand factor. In this study, once-weekly…”
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Increasing optimal coagulation factor dosing in the paediatric emergency department: Update to a quality improvement study by Kendel, Nicole E., Abdelghani, Eman, Stanek, Joseph R., Mitchell, Brockton, Dunn, Michael W., Rodriguez, Vilmarie, Dunn, Amy L.

“…Introduction Prompt, appropriate coagulation factor replacement according to injury and bleeding severity in persons with haemophilia is required to prevent…”
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Intracranial haemorrhage in children and adolescents with severe haemophilia A or B – the impact of prophylactic treatment by Andersson, Nadine G., Auerswald, Günter, Barnes, Chris, Carcao, Manuel, Dunn, Amy L., Fijnvandraat, Karin, Hoffmann, Marianne, Kavakli, Kaan, Kenet, Gili, Kobelt, Rainer, Kurnik, Karin, Liesner, Ri, Mäkipernaa, Anne, Manco‐Johnson, Marilyn J., Mancuso, Maria E., Molinari, Angelo C., Nolan, Beatrice, Perez Garrido, Rosario, Petrini, Pia, Platokouki, Helen E., Shapiro, Amy D., Wu, Runhui, Ljung, Rolf

“…Summary The discussion of prophylactic therapy in haemophilia is largely focused on joint outcomes. The impact of prophylactic therapy on intracranial…”
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Moderate-intensity aerobic exercise vs desmopressin in adolescent males with mild hemophilia A: a randomized trial by Kumar, Riten, Dunn, Amy L., Schneiderman, Jane E., Gonzales, Anne, Bouskill, Vanessa, Widener, Pamela, Stanek, Joseph, Pluthero, Fred G., Waller, Amanda, Tarango, Cristina, Ahuja, Sanjay, Kerlin, Bryce A., Kahr, Walter H.A., Rand, Margaret L., Lillicrap, David, Carcao, Manuel

“…Persons with mild hemophilia A (HA) may use intranasal desmopressin prior to sports participation. Desmopressin is expensive and can cause vomiting, headache,…”
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Radionuclide synovectomy for hemophilic arthropathy: a comprehensive review of safety and efficacy and recommendation for a standardized treatment protocol by Dunn, Amy L, Busch, Michael T, Wyly, J Bradley, Abshire, Thomas C

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Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1 by O'Mahony, Brian, Dunn, Amy L, Leavitt, Andrew D, Peyvandi, Flora, Ozelo, Margareth C, Mahlangu, Johnny, Peerlinck, Kathelijne, Wang, Jiaan-Der, Lowe, Gillian C, Tan, Chee Wee, Giermasz, Adam, Tran, Huyen, Khoo, Teh-Liane, Cockrell, Erin, Pepperell, Dominic, Chambost, Hervé, López Fernández, Maria Fernanda, Kazmi, Rashid, Majerus, Elaine, Skinner, Mark W, Klamroth, Robert, Quinn, Jennifer, Yu, Hua, Wong, Wing Yen, Robinson, Tara M, Pipe, Steven W

“…Severe hemophilia A (HA) negatively impacts health-related quality of life (HRQOL). We aimed to analyze HRQOL in adult men with severe HA without inhibitors…”
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Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A by Madan, Bella, Ozelo, Margareth C., Raheja, Priyanka, Symington, Emily, Quon, Doris V., Leavitt, Andrew D., Pipe, Steven W., Lowe, Gillian, Kenet, Gili, Reding, Mark T., Mason, Jane, Wang, Michael, von Drygalski, Annette, Klamroth, Robert, Shapiro, Susan, Chambost, Hervé, Dunn, Amy L., Oldenburg, Johannes, Chou, Sheng-Chieh, Peyvandi, Flora, Millar, Carolyn M., Osmond, Dane, Yu, Hua, Dashiell-Aje, Ebony, Robinson, Tara M., Mahlangu, Johnny

“…Valoctocogene roxaparvovec transfers a human factor (F)VIII coding sequence into hepatocytes of people with severe hemophilia A to provide bleeding protection…”
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International Prophylaxis Study Group (IPSG) haemophilia joint MRI scale version 2.0 by Lundin, Björn, Bakeer, Nihal, Dunn, Amy L, Gibikote, Sridhar, Keshava, Shyamkumar N, Ljung, Rolf C R, Papakonstantinou, Olympia, Pergantou, Helen, Strike, Karen, Drygalski, Annette von, Zhang, Ningning, Babyn, Paul, Dover, Saunya, Doria, Andrea S

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Improving 1-Deamino-8-D-Arginine Vasopressin (DDAVP) Challenges in Pediatric/Young Adult Patients With Bleeding Disorders: A Quality Improvement Study by Hallam, Michelle, Pruett, Amber, Biega, Charmaine, Miller, Haley, Davis, Tami, Casto, Linda, Dunn, Amy L., Rodriguez, Vilmarie

“…Background: Desmopressin (1-deamino-8-D-arginine vasopressin [DDAVP]) has demonstrated efficacy as a treatment option for patients with inherited bleeding…”
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Screening urinalysis demonstrates that haematuria is a frequent finding in persons with haemophilia treated at a paediatric haemophilia treatment centre by Davis, Kyle A., Stanek, Joseph R., Dunn, Amy L.

“…Introduction Haematuria is a recognized complication of haemophilia A and B (HA, HB). Adult persons with haemophilia (PWH) have a higher prevalence of renal…”
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Anti-Factor Xa–Based Monitoring of Unfractionated Heparin: Clinical Outcomes in a Pediatric Cohort by Saini, Surbhi, Folta, Ashley N., Harsh, Katherine L., Stanek, Joseph R., Dunn, Amy L., O'Brien, Sarah H., Kumar, Riten

“…To assess clinical outcomes in children treated with unfractionated heparin and monitored using an anti-factor Xa (Anti-FXa)-based nomogram. We also sought to…”
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A regional anticoagulation program improves safety and outcomes for both children and adults by Rodriguez, Vilmarie, Stanek, Joseph, Cua, Clifford L, Sankar, Amanda, Giver, Jean, Monda, Kay, Canini, Joan, Dunn, Amy L., Kerlin, Bryce A.

“…Background: Evidence-based anticoagulation programs usually serve a local, adult patient population. Here we report outcomes for a regional combined…”
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Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years by Pierce, Glenn F, Adediran, Megan, Diop, Saliou, Dunn, Amy L, El Ekiaby, Magdy, Kaczmarek, Radoslaw, Konkle, Barbara A, Pipe, Steven W, Skinner, Mark W, Valentino, Leonard A, Robinson, Fiona, Ampartzidis, Georgios, Martin, Jayson, Haffar, Assad

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