2025 (2 POSTS)
Sidhu MK, Miller B , Jensen I , Rabiner P. 2025. Abstract EE171: Conceptual framework for a de novo cost-effectiveness model in glycogen storage disease type Ia (GSDIa). Value in Health 28(6-Sup1):S93-S94; doi: 10.1016/j.jval.2025.04.463 .
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Topics: Health Economics and Outcomes Research (HEOR)
Duong T, Haselkorn T, Miller B , Coats J, Jensen I , Ward E, Wood M, Graham RJ, Servais L. 2025. A real-world analysis of the impact of X-linked myotubular myopathy on caregivers in the United States. Orphanet J Rare Dis 20(1):224. doi: 10.1186/s13023-025-03583-w . PMID: 40355957.
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Topics: Health Economics and Outcomes Research (HEOR)
2024 (9 POSTS)
Haselkorn T, Hughes W, Schara-Schmidt U, Lennox A, Roca A, Miller B , Jensen I , Solomon F, et al. 2024. Abstract PCR257: Real-world impact of x-linked myotubular myopathy (XLMTM) on caregivers in the United Kingdom (UK), Germany, and Spain. Value in Health 27(12-Sup):S557; doi: 10.1016/j.val.2024.10.3499 .
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Topics: Health Economics and Outcomes Research (HEOR)
Olaye A, Bean K, Velikanova R, Wolters S, Miller B , Jensen I , Pang F. Cost-effectiveness of atidarsagene autotemcel (ARSA-CEL) gene therapy for treating metachromatic leukodystrophy (MLD) in Ireland, Belgium, and the Netherlands as a part of the Beneluxa Initiative. Abstract EE693, ISPOR Europe 2024. Value in Health 27(12 Sup):S191; doi: 10.1016/j.jval.2024.10.973 . Barcelona, November 2024.
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Topics: Health Economics and Outcomes Research (HEOR) , Metachromatic leukodystrophy (MLD)
Pang F, Dean R, Jensen I , Bean K, Fields C, Miller B . The cost-effectiveness of atidarsagene autotemcel (ARSACEL) for the treatment of metachromatic leukodystrophy (MLD) in Spain. ISPOR Europe 2024, November 2024.
Topics: Health Economics and Outcomes Research (HEOR) , Metachromatic leukodystrophy (MLD)
Haselkorn T, Roca A, Miller B, Jensen I , Solomon F, Sanchez-de la Rosa R, Picart JM, Nascimento A. A real-world impact of X-linked myotubular myopathy (XLMTM) on caregivers in Spain. Presented at LXXVI Reunion Annual de la Sociedad Espanola de Nurologica, Valencia, Spain, 19–23 November 2024.
Topics: Health Economics and Outcomes Research (HEOR)
Pang F, Dean R, Jensen I , Bean K, Fields C, Miller B. The 18-effectiveness of atidarsagene autotemcel (ARSA-CEL) for the treatment of metachromatic leukodystrophy (MLD) in Spain. ISPOR Europe 2024, Barcelona, November 2024.