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Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort

BACKGROUND: ADVANCE (NCT01526785) presented an opportunity to obtain a more nuanced understanding of motor function changes in treatment-experienced children with Pompe disease receiving 4000L-production-scale alglucosidase alfa for 52 weeks. OBJECTIVE: To estimate minimal detectable change (MDC) an...

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Detalles Bibliográficos
Autores principales:	Duong, Tina, Kishnani, Priya S., An Haack, Kristina, Foster, Meredith C., Gibson, James B., Wilson, Catherine, Hahn, Si Houn, Hillman, Richard, Kronn, David, Leslie, Nancy D., Peña, Loren D.M., Sparks, Susan E., Stockton, David W., Tanpaiboon, Pranoot, Day, John W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2022
Materias:	Research Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9697057/ https://www.ncbi.nlm.nih.gov/pubmed/36214004 http://dx.doi.org/10.3233/JND-210784

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