{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T01:10:36Z","timestamp":1777857036689,"version":"3.51.4"},"reference-count":37,"publisher":"SAGE Publications","issue":"6","license":[{"start":{"date-parts":[[2022,9,1]],"date-time":"2022-09-01T00:00:00Z","timestamp":1661990400000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2022,9,1]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>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\u200a=\u200a 90).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Overall, participants aged\u200a&lt;\u200a2 years surpassed MDC at Week 52 (change [mean\u00b1standard deviation] 21.1\u00b114.1, MDC range 5.7\u201313.3, effect size 1.1), whereas participants aged\u22652 years did not attain this (change \u20130.9\u00b115.3, MDC range 10.8\u201325.2, effect size \u20130.03). In participants aged\u200a&lt;\u200a2 years, improvements surpassed the MDC for walkers (change 17.1\u00b113.3, MDC range 3.0\u20136.9, effect size 1.7), supported standers (change 35.2\u00b118.0, MDC range 5.9\u201313.7, effect size 1.8) and sitters (change 24.1\u00b112.1, MDC range 2.6\u20136.2, effect size 2.7). Age-independent MDC ranges were only attained by walkers (change 7.7\u00b112.3, MDC range 6.4\u201315.0, effect size 0.4) and sitters (change 9.9\u00b117.2, MDC range 3.3\u20137.7, effect size 0.9).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>These first GMFM-88 minimal-detectable-change estimates for alglucosidase alfa-treated Pompe disease offer utility for monitoring motor skills.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial registration:<\/jats:title>\n                    <jats:p>ClinicalTrials.gov; NCT01526785; Registered 6 February 2012; https:\/\/clinicaltrials.gov\/ct2\/show\/NCT01526785<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-210784","type":"journal-article","created":{"date-parts":[[2022,10,4]],"date-time":"2022-10-04T11:29:50Z","timestamp":1664882990000},"page":"713-730","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":3,"title":["Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort"],"prefix":"10.1177","volume":"9","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4619-9671","authenticated-orcid":false,"given":"Tina","family":"Duong","sequence":"first","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine, Stanford University School of Medicine, Palo Alto, CA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Priya S.","family":"Kishnani","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Duke University Medical Center, Durham, NC, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kristina","family":"An Haack","sequence":"additional","affiliation":[{"name":"Sanofi, Chilly-Mazarin, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Meredith C.","family":"Foster","sequence":"additional","affiliation":[{"name":"Sanofi, Cambridge, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"James B.","family":"Gibson","sequence":"additional","affiliation":[{"name":"Clinical and Metabolic Genetics, Dell Children\u2019s Medical Group, Austin, TX, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Catherine","family":"Wilson","sequence":"additional","affiliation":[{"name":"Sanofi, Cambridge, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Si Houn","family":"Hahn","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Medicine and Biochemical Genetics Program, Seattle Children\u2019s Hospital\/University of Washington, Seattle, WA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard","family":"Hillman","sequence":"additional","affiliation":[{"name":"University of Missouri Child Health, Columbia, MO, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"David","family":"Kronn","sequence":"additional","affiliation":[{"name":"Departments of Pathology and Pediatrics, New York Medical College, Valhalla, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nancy D.","family":"Leslie","sequence":"additional","affiliation":[{"name":"Division of Human Genetics, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Loren D.M.","family":"Pe\u00f1a","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Duke University Medical Center, Durham, NC, USA"},{"name":"Current address: Division of Human Genetics, Cincinnati Children\u2019s Hospital Medical Center, Cincinnati, OH, USA"},{"name":"Current address: University of Cincinnati College of Medicine, Cincinnati, OH, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Susan E.","family":"Sparks","sequence":"additional","affiliation":[{"name":"Sanofi, Cambridge, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"David W.","family":"Stockton","sequence":"additional","affiliation":[{"name":"Division of Genetic, Genomic, and Metabolic Disorders, Central Michigan University and Children\u2019s Hospital of Michigan, Detroit, MI, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pranoot","family":"Tanpaiboon","sequence":"additional","affiliation":[{"name":"Rare Disease Institute, Children\u2019s National Hospital, Washington, DC, USA"},{"name":"Current address: Division of Medical Genetics, Child Health Research Center, Torrance, CA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"John 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