Search Results - "Pinachyan, Karen"

A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient by Miesbach, Wolfgang, Mulders, Greta, Breederveld, Daan, Pinachyan, Karen, Le Quellec, Sandra, Pabinger, Ingrid

“…Adeno-associated virus (AAV)-based gene therapy for haemophilia has advanced substantially in the last 13 years; recently, three products have received…”
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Pharmacokinetic model-based assessment of factor IX prophylaxis treatment regimens in severe hemophilia B by Vandewalle, Björn, Castaman, Giancarlo, Álvarez-Román, Maria Teresa, Ettingshausen, Carmen Escuriola, Nemes, László, Tomic, Radovan, Martins, Paulo, Rodrigues, Joana F., Pinachyan, Karen

“…An important aspect of improving care for people with hemophilia B (HB) is developing optimal treatment strategies. Here we aimed to provide in-silico…”
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A multi-stakeholder collaborative approach to awareness, education and support needed in the haemophilia gene therapy patient journey – a report on insights from a European patient advisory board by O’Mahony, Brian, Fletcher, Simon, Baarslag, Manuel, Khair, Kate, Breederveld, Daan, Jansen, Nathalie, Pembroke, Luke, Pinachyan, Karen, Sivasubramaniyam, Sujan

“…Gene therapy presents a potentially transformational approach to haemophilia management. The patient journey for gene therapy is unlike that for other…”
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Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review by Ay, Cihan, Frenzel, Laurent, Pinachyan, Karen, Le Quellec, Sandra

“…Introduction With recent approval of the first two gene therapies for haemophilia A and B, educational materials about AAV‐based gene therapy are needed by the…”
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Classification of recombinant factor VIII products and implications for clinical practice: A systematic literature review by Ay, Cihan, Napolitano, Mariasanta, Hassoun, Abel, Tomic, Radovan, Martin, Cedric, Seifert, Wilfried, Pinachyan, Karen, Oldenburg, Johannes

“…Consensus over the definition of recombinant factor VIII (rFVIII) product classification in haemophilia A is lacking. rFVIII products are often classified as…”
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Analysis of pooled real-world data from Germany, Italy, and the United States of rVIII-SingleChain compared with standard- and long-acting FVIII products for prophylaxis of hemophilia A by Olivieri, Martin, Simpson, Mindy, Yan, Songkai, Fedorovsky, Johanna, Zhang, Xiang, Tomic, Radovan, Pinachyan, Karen, Mancuso, Maria Elisa

“…To evaluate real-world annualized bleeding rates (ABRs), dosing frequency, and factor consumption of four recombinant FVIII (rFVIII) products using pooled data…”
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Featured Cover by Miesbach, Wolfgang, von Drygalski, Annette, Smith, Clive, Sivamurthy, Krupa, Pinachyan, Karen, Bensen‐Kennedy, Debbie, Drelich, Douglass, Kulkarni, Roshni

“…The cover image is based on the Review The current challenges faced by people with hemophilia B by Wolfgang Miesbach et al., https://doi.org/10.1111/ejh.14135…”
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The current challenges faced by people with hemophilia B by Miesbach, Wolfgang, Drygalski, Annette, Smith, Clive, Sivamurthy, Krupa, Pinachyan, Karen, Bensen‐Kennedy, Debbie, Drelich, Douglass, Kulkarni, Roshni

“…Hemophilia B (HB) is a rare, hereditary disease caused by a defect in the gene encoding factor IX (FIX) and leads to varying degrees of coagulation deficiency…”
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Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial by Coppens, Michiel, Pipe, Steven W, Miesbach, Wolfgang, Astermark, Jan, Recht, Michael, van der Valk, Paul, Ewenstein, Bruce, Pinachyan, Karen, Galante, Nicholas, Le Quellec, Sandra, Monahan, Paul E, Leebeek, Frank W G, Castaman, Giancarlo, Crary, Shelley E, Escobar, Miguel, Gomez, Esteban, Haley, Kristina M, Hermans, Cedric R J R, Kampmann, Peter, Kazmi, Rashid, Key, Nigel S, Klamroth, Robert, Konkle, Barbara A, Kruse-Jarres, Rebecca, Lattimore, Susan, Lemons, Richard, Meijer, Karina, O'Connell, Niamh, Quon, Doris V, Raheja, Priyanka, Symington, Emily, Verhamme, Peter, Visweshwar, Nathan, von Drygalski, Annette, Wang, Michael, Wheeler, Allison P, White, Shanna, Young, Guy

“…Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B treatment, was shown to be superior to treatment with continuous prophylactic…”
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Assessing cancer drugs for reimbursement: methodology, relationship between effect size and medical need by de Sahb-Berkovitch, Rima, Woronoff-Lemsi, Marie-Christine, Molimard, Mathieu

“…Reimbursement is assessed by the Transparency Commission from the Health Authority (HAS) using a medical benefit (SMR) score that gives access to…”
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Collections of human biological samples for scientific purposes. Why do current regulation need to be clarified and how? by Deplanque, Dominique, Birraux, Guillaume, Bertoye, Pierre-Henri, Postaire, Eric

“…The collection of human biological samples is of major importance for future research in France and Europe. In recent years, new regulatory procedures have…”
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