Search Results - James Signorovitch
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Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis by Shannon Grabich, Brian Ung, Aalok Nadkar, Kathryn DeYoung, James Signorovitch, Aravindhan Veerapandiyan
Published 2025-07-01Aim: Phosphorodiamidate morpholino oligomers (PMOs) are exon-skipping therapies administered through once-weekly intravenous infusions used to treat Duchenne muscular dystrophy (DMD). This study assessed treatment patterns among patients with DMD receiving PMOs using administrative claims data wh...
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Healthcare Utilization and Costs Among US Adolescents With Alopecia Areata by Markqayne Ray, Elyse Swallow, Kavita Gandhi, Christopher Carley, Vanja Sikirica, Travis Wang, Nicolae Done, James Signorovitch, Arash Mostaghimi
Published 2022-07-01# Background Alopecia areata (AA) is an autoimmune disease of hair loss affecting people of all ages. Alopecia totalis (AT) and universalis (AU) involve scalp and total body hair loss, respectively. AA significantly affects quality of life, but evidence on the economic burden in adolescents is limit...
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Temporal trends in vaccination and antibiotic use among young children in the United States, 2000–2019 by Amanda L. Eiden, Qing Liu, Yoonyoung Choi, Yan Song, Gary S. Marshall, Nicolae Done, Travis Wang, Goran Bencina, James Signorovitch
Published 2025-01-01Abstract Objective: Routine immunization programs may reduce antibiotic use, but few studies have comprehensively examined their impact on antibiotic utilization. We aimed to explore temporal trends in vaccination and antibiotic use among young children in the United States. Design: Ecological s...
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Comparative Effectiveness of Second-Line Targeted Therapies for Metastatic Renal Cell Carcinoma: A Systematic Review and Meta-Analysis of Real-World Observational Studies. by Daniel Y Heng, James Signorovitch, Elyse Swallow, Nanxin Li, Yichen Zhong, Paige Qin, Daisy Y Zhuo, Xufang Wang, Jinhee Park, Sotirios Stergiopoulos, Christian Kollmannsberger
Published 2014-01-01<h4>Objective</h4>The optimal sequencing of targeted therapies for metastatic renal cell carcinoma (mRCC) is unknown. Observational studies with a variety of designs have reported differing results. The objective of this study is to systematically summarize and interpret the published re...
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Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy by 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, Laurant Servais, Eugenio Mercuri, Michela Guglieri, Volker Straub, Imelda de Groot, Deborah Ridout, PRO-DMD-01 study investigators, Association Française contre les Myopathies, The NorthStar Clinical Network, Craig McDonald
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Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy. by 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, Laurant Servais, Eugenio Mercuri, Michela Guglieri, Volker Straub, Imelda de Groot, Deborah Ridout, PRO-DMD-01 study investigators, Association Française contre les Myopathies, NorthStar Clinical Network, Craig McDonald
Published 2025-01-01The North Star Ambulatory Assessment (NSAA) is a widely used functional endpoint in drug development for ambulatory patients with Duchenne muscular dystrophy (DMD). Accurately predicting NSAA total score trajectories is important for designing randomized trials for novel therapies in DMD and for con...
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