Citations (58)
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Alexa C. Klimchak, Lauren E. Sedita, Louise R. Rodino-Klapac, Jerry R. Mendell, Craig M. McDonald, Katherine L. Gooch & Daniel C. Malone. (2023) Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States. Journal of Market Access & Health Policy 11:1.
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Alexa C. Klimchak, Lauren E. Sedita, Katherine L. Gooch & Daniel C. Malone. (2023) Assessing the impact of single or short-term administration on a therapy’s cost-effectiveness: a hypothetical disease-agnostic model. Journal of Medical Economics 26:1, pages 594-602.
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Mondher Toumi, Omar Dabbous, Samuel Aballéa, Michael F. Drummond, Johann-Matthias Graf von der Schulenburg, Daniel C. Malone, Peter J. Neumann, Sean D. Sullivan & Sean Tunis. (2023) Recommendations for economic evaluations of cell and gene therapies: a systematic literature review with critical appraisal. Expert Review of Pharmacoeconomics & Outcomes Research 23:5, pages 483-497.
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Samira Saberian, Philippa Rowan, Florence Hammes, Priya Patel, Fernando Fernandez-Cortes, Katharina Buesch & Igor Beitia Ortiz de Zarate. (2022) Burden of illness of aromatic L-amino acid decarboxylase deficiency: a survey of physicians in Southern Europe. Current Medical Research and Opinion 38:7, pages 1115-1123.
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Rebecca Dean, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Thomas Wiesner, Daniel C. Malone, Matthias Bischof, Walter Toro & Omar Dabbous. (2021) An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). Journal of Market Access & Health Policy 9:1.
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Hongbo Yang, Yanni Hao, Xinglei Chai, Cynthia Z. Qi & Eric Q. Wu. (2020) Estimation of total costs in patients with relapsed or refractory diffuse large B-cell lymphoma receiving tisagenlecleucel from a US hospital’s perspective. Journal of Medical Economics 23:9, pages 1016-1024.
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Martin Connock, Lazaros Andronis, Peter Auguste, Claude Dussart & Xavier Armoiry. (2020) Will the US$5 million onasemnogene abeparvosec treatment for spinal muscular atrophy represent ‘value for money’ for the NHS? A rapid inquiry into suggestions that it may be cost-effective. Expert Opinion on Biological Therapy 20:7, pages 823-827.
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Lisa Belter, Rosángel Cruz, Sierra Kulas, Emily McGinnis, Omar Dabbous & Jill Jarecki. (2020) Economic burden of spinal muscular atrophy: an analysis of claims data. Journal of Market Access & Health Policy 8:1.
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Samuel Aballéa, Katia Thokagevistk, Rimma Velikanova, Steven Simoens, Lieven Annemans, Fernando Antonanzas, Pascal Auquier, Clément François, Frank-Ulrich Fricke, Daniel Malone, Aurélie Millier, Ulf Persson, Stavros Petrou, Omar Dabbous, Maarten Postma & Mondher Toumi. (2020) Health economic evaluation of gene replacement therapies: methodological issues and recommendations. Journal of Market Access & Health Policy 8:1.
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