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

    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

    Published 2025-01-01 Get full text
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    Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy.

    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 2025-01-01

    The 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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