Publications

Francesco Muntoni, James Signorovitch, Nathalie Goemans, Adnan Y. Manzur, Nicolae Done, Gautam Sajeev, Jiayang Li, Hanane Akbarnejad, Aarushi Sharma, Susan J. Ward, Erik H. Niks, Laurent Servais, Eugenio Mercuri, Michela Guglieri, Volker Straub, Imelda de Groot, Deborah Ridout, Craig McDonald (2025). Predicting North Star Ambulatory Assessment trajectories in Duchenne muscular dystrophy: A mixed-effects modeling approach. PLoS ONE.

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Francesco Muntoni, James Signorovitch, Gautam Sajeev, Nicolae Done, Zhiwen Yao, Nathalie Goemans, Craig McDonald, Eugenio Mercuri, Erik H. Niks, Brenda Wong, Krista Vandenborne, Volker Straub, Imelda J. M. de Groot, Cuixia Tian, Adnan Manzur, Ibrahima Dieye, Henry Lane, Susan J. Ward, Laurent Servais (2024). Minimal detectable change and meaningful change in motor function in Duchenne muscular dystrophy. PLoS ONE.

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Joel Iff, Nicolae Done, Edward Tuttle, Yi Zhong, Fangzhou Wei, Basil T. Darras, Craig M. McDonald, Eugenio Mercuri, Francesco Muntoni (2024). Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls. Muscle Nerve.

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James Signorovitch, Jialu Zhang, David Brown, Preston Dunnmon, Liang Xiu, Nicolae Done, Kristen Hsu, Yolanda Barbachano, Isabelle Lousada (2024). Pathway for Development and Validation of Multi-domain Endpoints for Amyloid Light Chain (AL) Amyloidosis. TIRS.

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Wei Gao, Arash Mostaghimi, Kavita Gandhi, Nicolae Done, Markqayne Ray, James Signorovitch, Elyse Swallow, Christopher Carley, Travis Wang, Vanja Sikirica (2023). Patient characteristics associated with all-cause healthcare costs of alopecia areata in the United States. J Med Econ.

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Chafic Karam, Duncan Brown, Min Yang, Nicolae Done, Jing Jing Zhu, Alexandra Greatsinger, Ana Bozas, Montserrat Vera-Llonch, James Signorovitch (2022). Long-term treatment effects of inotersen on health-related quality of life in patients with hATTR amyloidosis with polyneuropathy: Analysis of the open-label extension of the NEURO-TTR trial. Muscle Nerve.

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Kim A. Papp, Ahmed M. Soliman, Nicolae Done, Christopher Carley, Esteban Lemus Wirtz, Luis Puig (2021). Deterioration of Health-Related Quality of Life After Withdrawal of Risankizumab Treatment in Patients with Moderate-to-Severe Plaque Psoriasis: A Machine Learning Predictive Model. Dermatol Ther.

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Nicolae Done, Bradley Herring, Tim Xu (2019). The effects of global budget payments on hospital utilization in rural Maryland. HSR.

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Lauren H. Nicholas, Nicolae Done, Micah Baum (2018). Lifetime job demands and later life disability. J Econ Ageing.

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Nicolae Done, Amanda M. Roy, Yingzhe Yuan, Steven D. Pizer, Adam J. Rose, Julia C. Prentice (2018). Guideline-concordant initiation of oral anticoagulant therapy for stroke prevention in older veterans with atrial fibrillation eligible for Medicare Part D. HSR.

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Nicolae Done, Dennis H. Oh, Martin A. Weinstock, John D. Whited, George L. Jackson, Heather A. King, Sara B. Peracca, A. Rani Elwi, Julia C. Prentice (2018). VA Telederm study: protocol for a stepped-wedge cluster randomised trial to compare access to care for a mobile app versus a workstation-based store-and-forward teledermatology process. BMJ Open.

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Joy Z. Zhou, Benjamin T. Lemelman, Nicolae Done, Macey L. Henderson, Alexandra Macmillan, David H. Song, Amir H. Dorafshar (2018). Social Media and the Dissemination of Research: Insights from the Most Widely Circulated Articles in Plastic Surgery. Plast Reconstr Surg.

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Jong Chul Rhee, Nicolae Done, Gerard F. Anderson (2015). Long-Term Care Insurance in Korea, Japan, and Germany: Lessons for Developing Countries. Health Policy.

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William C. Hsiao, Anna G. Knight, Steven Kappel, Nicolae Done (2011). What Other States Can Learn From Vermont's Bold Experiment: Embracing A Single-Payer Health Care Financing System. Health Aff.

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