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Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls.
Muntoni, Francesco; Signorovitch, James; Sajeev, Gautam et al.
2022 • In Neuromuscular Disorders, 32 (4), p. 271-283

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MuntoniNMD2022.pdf

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