{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,17]],"date-time":"2026-07-17T06:07:53Z","timestamp":1784268473397,"version":"3.55.0"},"reference-count":15,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2024,4,8]],"date-time":"2024-04-08T00:00:00Z","timestamp":1712534400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100015362","name":"Amicus Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100015362","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2024,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Late\u2010onset Pompe disease (LOPD) is characterized by a progressive myopathy resulting from a deficiency of acid \u03b1\u2010glucosidase enzyme activity. Enzyme replacement therapy has been shown to be effective, but long\u2010term treatment results vary. Avalglucosidase alfa demonstrated non\u2010inferiority to alglucosidase alfa in a phase 3 study, allowing in France compassionate access for advanced LOPD patients unresponsive to alglucosidase alfa.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Data from the French Pompe registry were analyzed for patients who benefited from a switch to avalglucosidase alfa with at least 1\u2009year of follow\u2010up. Respiratory (forced vital capacity [FVC]) and motor functions (Six\u2010Minute Walk Test [6MWT]) were assessed before and 1\u2009year after switching. Individual changes in FVC and 6MWT were expressed as slopes and statistical analyses were performed to compare values.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Twenty\u2010nine patients were included (mean age 56\u2009years, 11\u2009years of prior treatment). The FVC and 6MWT values remained stable. The individual analyses showed a stabilization of motor worsening: \u20131\u2009m\/year on the 6MWT after the switch versus \u201363\u2009m\/year the year before the switch (i.e., a worsening of 33%\/year before vs. an improvement of 3%\/year later). Respiratory data were not statistically different.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>At the group level, gait parameters improved slightly with a stabilization of previous worsening, but respiratory parameters showed limited changes. At the individual level, results were discordant, with some patients with a good motor or respiratory response and some with further worsening.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Switching to avalglucosidase alfa demonstrated varied responses in advanced LOPD patients with failing alglucosidase alfa therapy, with a general improvement in motor stabilization.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ene.16292","type":"journal-article","created":{"date-parts":[[2024,4,8]],"date-time":"2024-04-08T10:39:50Z","timestamp":1712572790000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Real\u2010life effectiveness 1 year after switching to avalglucosidase alfa in late\u2010onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6654-8090","authenticated-orcid":false,"given":"C\u00e9line","family":"Tard","sequence":"first","affiliation":[{"name":"Inserm, CHU Lille, U1172 \u2013 LilNCog \u2013 Lille Neuroscience and Cognition University of Lille  Lille France"},{"name":"Centre de R\u00e9f\u00e9rence des Maladies NeuroMusculaires Nord \u2013 Est \u2013 Ile\u2010de\u2010France, Filnemus  Garches France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fran\u00e7oise","family":"Bouhour","sequence":"additional","affiliation":[{"name":"Centre de R\u00e9f\u00e9rence des Maladies NeuroMusculaires PACA R\u00e9union Rh\u00f4ne Alpes, Filnemus  Marseille France"},{"name":"Service ENMG\/Pathologies Neuromusculaires Hospices Civils de Lyon  Lyon France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maud","family":"Michaud","sequence":"additional","affiliation":[{"name":"Centre de R\u00e9f\u00e9rence 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