{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T01:13:06Z","timestamp":1777857186492,"version":"3.51.4"},"reference-count":14,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2023,12,27]],"date-time":"2023-12-27T00:00:00Z","timestamp":1703635200000},"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":[[2024,3,5]]},"abstract":"<jats:p>In the COMET trial of patients with late-onset Pompe disease, greater improvement in upright forced vital capacity (FVC) % predicted was observed with avalglucosidase alfa (AVA) vs alglucosidase alfa (ALGLU) (estimated treatment difference: 2.43%). The pre-specified mixed model repeated measures (MMRM) analysis demonstrated non-inferiority of AVA ( P\u200a=\u200a0.0074) and narrowly missed superiority ( P\u200a=\u200a0.063; 95% CI: \u20130.13\u20134.99). We report superiority of AVA in two post-hoc analyses that account for an extreme outlier participant with low FVC and severe chronic obstructive pulmonary disease at baseline: MMRM excluding the outlier ( P\u200a=\u200a0.013) and non-parametric analysis of all data with repeated measures analysis of covariance ( P\u200a=\u200a0.019).<\/jats:p>","DOI":"10.3233\/jnd-230175","type":"journal-article","created":{"date-parts":[[2023,12,29]],"date-time":"2023-12-29T12:07:21Z","timestamp":1703851641000},"page":"369-374","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa"],"prefix":"10.1177","volume":"11","author":[{"given":"Matthias","family":"Boentert","sequence":"first","affiliation":[{"name":"Department of Neurology and Institute of Translational Neurology, M\u00fcnster University Hospital, M\u00fcnster, Germany"},{"name":"Department of Medicine, UKM-Marienhospital Steinfurt, Steinfurt, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Emmanuelle Salort","family":"Campana","sequence":"additional","affiliation":[{"name":"Referral Centre for Neuromuscular Diseases and ALS, H\u00f4pital La Timone, Marseille, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shahram","family":"Attarian","sequence":"additional","affiliation":[{"name":"Referral Centre for Neuromuscular Diseases and ALS, H\u00f4pital La Timone, Marseille, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jordi","family":"Diaz-Manera","sequence":"additional","affiliation":[{"name":"Newcastle University Centre for Life, Newcastleupon Tyne, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mazen M.","family":"Dimachkie","sequence":"additional","affiliation":[{"name":"University of Kansas Medical Center, Department of Neurology, Kansas City, KS, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Magali","family":"Periquet","sequence":"additional","affiliation":[{"name":"Sanofi, Berlin, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nathan","family":"Thibault","sequence":"additional","affiliation":[{"name":"Sanofi, Cambridge, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patrick","family":"Miossec","sequence":"additional","affiliation":[{"name":"Sanofi, Paris, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tianyue","family":"Zhou","sequence":"additional","affiliation":[{"name":"Sanofi, Cambridge, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kenneth I.","family":"Berger","sequence":"additional","affiliation":[{"name":"NYU Grossman School of Medicine, New York, NY, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2023,12,27]]},"reference":[{"key":"ref001","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)61555-X"},{"key":"ref002","doi-asserted-by":"publisher","DOI":"10.21037\/atm.2019.07.24"},{"key":"ref003","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000004711"},{"key":"ref004","unstructured":"NEXVIAZYME (avalglucosidase alfa-ngpt) for injection, for intravenous use. [Prescribing Information] Cambridge, MA: Genzyme Corporation, August 2021. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2021\/761194s000lbl.pdf"},{"key":"ref005","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00241-6"},{"key":"ref006","doi-asserted-by":"publisher","DOI":"10.1021\/bc400365a"},{"key":"ref007","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2009.37"},{"key":"ref008","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201105-0840OC"},{"key":"ref009","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-8-160"},{"key":"ref010","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2023.03.2304"},{"key":"ref011","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.01.013"},{"key":"ref012","doi-asserted-by":"publisher","DOI":"10.7326\/0003-4819-155-3-201108020-00008"},{"key":"ref013","unstructured":"Global Initiative for Chronic Obstructive Lung Disease. Global Strategy fo the Diagnosis, Management, and Prevention of COPD (2023). Available at https:\/\/goldcopd.org\/2023-gold-report-2\/. Accessed June 25, 2023."},{"key":"ref014","doi-asserted-by":"publisher","DOI":"10.1080\/03610926.2015.1014106"}],"container-title":["Journal of Neuromuscular Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.3233\/JND-230175","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.3233\/JND-230175","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.3233\/JND-230175","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T23:52:38Z","timestamp":1777506758000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.3233\/JND-230175"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12,27]]},"references-count":14,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2024,3,5]]}},"alternative-id":["10.3233\/JND-230175"],"URL":"https:\/\/doi.org\/10.3233\/jnd-230175","relation":{},"ISSN":["2214-3599","2214-3602"],"issn-type":[{"value":"2214-3599","type":"print"},{"value":"2214-3602","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,12,27]]}}}