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2022 | 1 |
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Development of a model-based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy.
CPT Pharmacometrics Syst Pharmacol. 2022 Mar;11(3):318-332. doi: 10.1002/psp4.12753. Epub 2022 Jan 3.
CPT Pharmacometrics Syst Pharmacol. 2022.
PMID: 34877803
Free PMC article.
The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures.
Henricson EK, Abresch RT, Cnaan A, Hu F, Duong T, Arrieta A, Han J, Escolar DM, Florence JM, Clemens PR, Hoffman EP, McDonald CM; CINRG Investigators.
Henricson EK, et al.
Muscle Nerve. 2013 Jul;48(1):55-67. doi: 10.1002/mus.23808. Epub 2013 May 6.
Muscle Nerve. 2013.
PMID: 23649481
Free PMC article.
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