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Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

OBJECTIVE: To evaluate the suitability of real-world data (RWD) and natural history data (NHD) for use as external controls in drug evaluations for ambulatory Duchenne muscular dystrophy (DMD). METHODS: The consistency of changes in the 6-minute walk distance (Δ6MWD) was assessed across multiple cli...

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Detalles Bibliográficos
Autores principales:	Goemans, Nathalie, Signorovitch, James, Sajeev, Gautam, Yao, Zhiwen, Gordish-Dressman, Heather, McDonald, Craig M., Vandenborne, Krista, Miller, Debra, Ward, Susan J., Mercuri, Eugenio
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Lippincott Williams & Wilkins 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7538219/ https://www.ncbi.nlm.nih.gov/pubmed/32611643 http://dx.doi.org/10.1212/WNL.0000000000010170

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