2025
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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Publication: Manuscripts
Nordyke RJ , Motyka J, Patterson JA. 2025. The association of 340B program drug margins with covered entity characteristics. Inquiry 62(Jan-Dec):469580251324051; doi: 10.1177/00469580251234051 . PMID: 4012322.
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Publication: Manuscripts
Shafrin J, Wang S, Sharma K, Spurrier K, Nordyke RJ . 2025. Will the Institute for Clinical and Economic Review’s shared savings approach decrease value-based prices most for the most severe diseases? Value Health 28(1):25-30; doi: 10.1016/j.val.2024.09.002 . PMID: 39357669.
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Publication: Manuscripts
2024
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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Publication: Abstracts and Presentations
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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Publication: Abstracts and Presentations
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.
Publication: Abstracts and Presentations
Kelsh M , Bylsma LC, Jiang X, Movva N , Sacks NC . To be an orphan disease or not to be: An evaluation of prevalence estimates for cancers near the orphan status threshold and implications for orphan drug planning. International Society for Pharmacoepidemiology, 40th Annual Meeting, Berlin, Germany, August 2024.
Publication: Abstracts and Presentations
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.
Publication: Abstracts and Presentations
Seo S, Healey BE, McLin R, Sacks NC , Benson CJ, Citrome L. 2024. Impact of demographics and insurance coverage on schizophrenia treatment and healthcare resource utilization within an integrated healthcare system. Neuropsychiatr Dis Treat 20(Sept 26):1837–1848; doi: 10.2147/NDT.S473492 . PMID: 39351585.
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Publication: Manuscripts
DiBiase JF, Scharnetzki E, Edelman E, Reed EK, Helbig P, Rueter J, Miesfeldt S, Frankenfeld CL , et al. 2024. Socioeconomic and urban-rural disparities in genome-matched treatment receipt and survival after genomic tumor testing. JNCI Cancer Spectr 8(5):pkae090; doi: 10.1093/jncics/pkae090 . PMID: 39312685.
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