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Showing 1 - 13 of 163

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  • Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy

    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

    published 27 Jun 2025

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  • Characterization of patients with Duchenne muscular dystrophy across previously developed health states

    Francesco Muntoni, Nathalie Goemans, Nate Posner, James Signorovitch, Michaela Johnson, Chujun He, Patricia Dorling, Katherine Beaverson, Jose Alvir, Matthias Mahn, Susan J. Ward, Craig M. McDonald, Eugenio Mercuri, the PRO-DMD-01 study, the Imaging DMD study, iMDEX, the collaborative Trajectory Analysis Project (cTAP)

    published 30 Oct 2024

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  • Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study

    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, PRO-DMD-01 study investigators , Association Française contre les Myopathies , The UK NorthStar Clinical Network , ImagingDMD investigators , cTAP

    published 10 Jul 2024

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