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, Study Investigators Pro-Dmd-01, Association Française contre les Myopathies, The NorthStar Clinical Network and Craig McDonald

PLOS ONE, 2025, vol. 20, issue 6, 1-19

Abstract: 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 contextualizing outcomes, especially over longer-term follow-up (>18 months) when placebo-controlled studies are infeasible. We developed a prognostic model for NSAA total score trajectories over at most 5 years of follow-up for patients with DMD aged 4 to

Date: 2025
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Persistent link: https://EconPapers.repec.org/RePEc:plo:pone00:0325736

DOI: 10.1371/journal.pone.0325736

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