2025 (1 POST)
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 .
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Topics: Health Economics and Outcomes Research (HEOR)
2024 (8 POSTS)
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, November 2024.
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Topics: Health Economics and Outcomes Research (HEOR) , Metachromatic leukodystrophy (MLD)
Haselkorn T, Hughes W, Lennox A, Schara-Schmidt U, Roca A, Miller B, Jensen I , et al. Real-world impact of X-linked myotubular myopathy (XLMTM) on caregivers in the United Kingdom (UK), Germany, and Spain. Abstract PCR257 , ISPOR EU 2024 (Top 5% Finalist).
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Topics: Health Economics and Outcomes Research (HEOR)
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, 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, November 2024.