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Lea Wiedmann & John Cairns. (2023) Review of economic modeling evidence from NICE appraisals of rare disease treatments for spinal muscular atrophy. Expert Review of Pharmacoeconomics & Outcomes Research 23:5, pages 469-482.
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Adam B Smith, Andria Hanbury, Jennifer A Whitty, Igor Beitia Ortiz de Zarate, Florence Hammes, Gérard de Pouvourville & Katharina Buesch. (2022) A Discrete Choice Experiment to Derive Health Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency in France. Patient Related Outcome Measures 13, pages 21-30.
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Lorna L. Freath, Alistair S. Curry, David M. W. Cork, Ivana F. Audhya & Katherine L. Gooch. (2022) QALYs and ambulatory status: societal preferences for healthcare decision making. Journal of Medical Economics 25:1, pages 888-893.
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Adam B Smith, Andria Hanbury, Igor Beitia Ortiz de Zarate, Florence Hammes, Gerard de Pouvourville & Katharina Buesch. (2021) Eliciting Health State Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency: A Vignette Study in France. Patient Related Outcome Measures 12, pages 237-246.
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Adam B Smith, Andria Hanbury, Jennifer A Whitty & Katharina Buesch. (2021) A Discrete Choice Experiment to Derive Health Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. Patient Related Outcome Measures 12, pages 97-106.
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Maria Stefanova Kamusheva & Maria Jordanova Dimitrova. (2020) Clinical and economic assessment of nusinersen: the Bulgarian perspective. Expert Opinion on Orphan Drugs 8:10, pages 403-415.
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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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Articles from other publishers (13)
Michela Meregaglia, Elena Nicod & Michael Drummond. (2022) The estimation of health state utility values in rare diseases: do the approaches in submissions for NICE technology appraisals reflect the existing literature? A scoping review. The European Journal of Health Economics 24:7, pages 1151-1216.
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Renalli Manuella Rodrigues Alves, Vanessa Van Der Linden & Lívia Barbosa de Andrade. (2023) Comprehensive assessment model for patients with spinal muscular atrophy: proposal of tools for clinical practice and real-world studies. Revista da Associação Médica Brasileira 69:3, pages 377-379.
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Elena Nicod, Andrew J Lloyd, Thomas Morel, Michela Meregaglia, Sheela Upadhyaya, Amanda Whittal, Karen Facey & Michael Drummond. (2022) Improving Interpretation of Evidence Relating to Quality of Life in Health Technology Assessments of Rare Disease Treatments. The Patient - Patient-Centered Outcomes Research 16:1, pages 7-17.
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Jiahao Hu, Lin Zhu, Han Bao, Yuhan Liu, Huanping Xing, Qi Kang & Chunlin Jin. (2022) Utility estimations of different health states of patients with type I, II, and III spinal muscular atrophy in China: A mixed approach study with patient and proxy-reported data. Frontiers in Public Health 10.
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Min Yang, Hiroyuki Awano, Satoru Tanaka, Walter Toro, Su Zhang, Omar Dabbous & Ataru Igarashi. (2022) Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy. Advances in Therapy 39:5, pages 1915-1958.
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C. Simone Sutherland, Pollyanna Hudson, Stephen Mitchell & Noman Paracha. (2021) Systematic Literature Review to Identify Utility Values in Patients with Spinal Muscular Atrophy (SMA) and Their Caregivers. PharmacoEconomics 40:S1, pages 39-67.
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Adam B. Smith, Andria Hanbury & Katharina Buesch. (2021) Eliciting health state utilities for Aromatic L-amino Acid Decarboxylase (AADC) deficiency: a UK vignette study. Journal of Patient-Reported Outcomes 5:1.
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Paul Gissen, Nicola Specchio, Andrew Olaye, Mohit Jain, Thomas Butt, Wrik Ghosh, Benjamin Ruban-Fell, Annabel Griffiths, Charlotte Camp, Zlatko Sisic, Christoph Schwering, Eva Wibbeler, Marina Trivisano, Laura Lee, Miriam Nickel, Amanda Mortensen & Angela Schulz. (2021) Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2). Orphanet Journal of Rare Diseases 16:1.
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Sophy TF Shih, Michelle Anne Farrar, Veronica Wiley & Georgina Chambers. (2021) Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis. Journal of Neurology, Neurosurgery & Psychiatry 92:12, pages 1296-1304.
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Andrew Lloyd, Daniel Aggio, Ted L. Slocomb, Jun Lee, Alan H. Beggs & Deborah A. Bilder. (2021) Estimation of the Quality-of-Life Impact of X-Linked Myotubular Myopathy. Journal of Neuromuscular Diseases 8:6, pages 1047-1061.
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Mette Kruijsbergen, Carin D Schröder, Marjolijn Ketelaar, W Ludo Pol, Inge Cuppen, Annette Geest, Fay‐Lynn Asselman, Maarten J Fischer, Johanna M A Visser‐Meily & Marijke C Kars. (2021) Parents’ perspectives on nusinersen treatment for children with spinal muscular atrophy. Developmental Medicine & Child Neurology 63:7, pages 816-823.
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Lisa Belter, Rosángel Cruz & Jill Jarecki. (2020) Quality of life data for individuals affected by spinal muscular atrophy: a baseline dataset from the Cure SMA Community Update Survey. Orphanet Journal of Rare Diseases 15:1.
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. (2019) QOL burden substantial in spinal muscular atrophy. PharmacoEconomics & Outcomes News 840:1, pages 23-23.
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