Main Model Specifications.

<div><p>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 therapie...

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المؤلف الرئيسي:	Francesco Muntoni (228210) (author)
مؤلفون آخرون:	James Signorovitch (672562) (author), Nathalie Goemans (503769) (author), Adnan Y. Manzur (7340012) (author), Nicolae Done (10751202) (author), Gautam Sajeev (6810248) (author), Jiayang Li (449346) (author), Hanane Akbarnejad (21615534) (author), Aarushi Sharma (5216615) (author), Susan J. Ward (3018144) (author), Erik H. Niks (674893) (author), Laurant Servais (21615537) (author), Eugenio Mercuri (289290) (author), Michela Guglieri (489528) (author), Volker Straub (251869) (author), Imelda de Groot (4399726) (author), Deborah Ridout (9096296) (author), Craig McDonald (2615428) (author)
منشور في:	2025
الموضوعات:	Medicine Genetics Environmental Sciences not elsewhere classified Biological Sciences not elsewhere classified Mathematical Sciences not elsewhere classified Chemical Sciences not elsewhere classified duchenne muscular dystrophy designing randomized trials 69 %) relative (> 18 months body mass index nsaa total score ambulatory motor function quartile range 1 dmd aged 4 div >< p daily corticosteroid regimens 5 nsaa units 6 years ). index year dmd ). initially ambulatory 6 years 5 years 16 years uz leuven steroid type run velocity receiving prednisone receiving corticosteroids prediction errors novel therapies modifying therapies meter walk mean age longitudinal data genotype class floor velocity drug development dmd </ discuss applications data source cureduchenne ), controlled studies contextualizing outcomes among n ambulatory patients 31 %).
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Main Model Specifications.

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