Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort

Autor:	Tina, Duong, Priya S, Kishnani, Kristina, An Haack, Meredith C, Foster, James B, Gibson, Catherine, Wilson, Si Houn, Hahn, Richard, Hillman, David, Kronn, Nancy D, Leslie, Loren D M, Peña, Susan E, Sparks, David W, Stockton, Pranoot, Tanpaiboon, John W, Day
Rok vydání:	2022
Předmět:	Cohort Studies Neurology Glycogen Storage Disease Type II Motor Skills Humans Enzyme Replacement Therapy Neurology (clinical) Child
Zdroj:	Journal of Neuromuscular Diseases. 9:713-730
ISSN:	2214-3602 2214-3599
Popis:	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) and effect size on Gross Motor Function Measure-88 (GMFM-88) after 52 weeks of 4000L alglucosidase alfa (complete data N = 90). Methods: The GMFM-88 mean total % score changes, MDC, and effect size were analyzed post hoc by Pompe Motor Function Level at enrollment, age groups at enrollment, and fraction of life on pre-study 160L-production-scale alglucosidase alfa. Results: Overall, participants aged
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::61506990a232a0cdf6bf04e87c605f31 https://doi.org/10.3233/jnd-210784 Zobrazit plný text záznamu