Publication Topic

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.

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.

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).

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.

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, 40^th Annual Meeting, Berlin, Germany, August 2024.

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.

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.

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.

Bean K, Jones SA, Chakrapani A, Vijay S, Wu T, Church H, … Jensen I, et al. 2024. Exploring the cost-effectiveness of newborn screening for metachromatic leukodystrophy (MLD) in the UK. Int J Neonatal Screen 10(3):45; doi: 10.3390/ijns10030045.

Chew DS, Sacks NC, Emden MR, Cyr PL, Sherwood R, Pokorney SD. 2024. Catheter ablation for supraventricular tachycardia and health resource utilization and expenditures: A propensity-matched cohort study. Int J Cardiol 403(May 15):131831; doi: 10.1016/j.ijcard.2024.131831. PMID: 38331201.