{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,28]],"date-time":"2026-07-28T12:02:31Z","timestamp":1785240151529,"version":"3.55.0"},"reference-count":31,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2026,7,16]],"date-time":"2026-07-16T00:00:00Z","timestamp":1784160000000},"content-version":"vor","delay-in-days":15,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T00:00:00Z","timestamp":1782864000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"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\/501100006044","name":"Astellas Pharma Europe","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100006044","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":[[2026,7]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:label\/>\n                    <jats:p>Late\u2010onset Pompe disease (LOPD) is a progressive myopathy. Enzyme replacement therapy is effective, but long\u2010term outcomes vary. Avalglucosidase alfa, shown to be non\u2010inferior to alglucosidase alfa in a phase 3 trial, became available in France through compassionate use for patients with insufficient response to alglucosidase alfa.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Data from the French Pompe registry were analyzed for patients who switched to avalglucosidase alfa with at least 1\u2009year of follow\u2010up. Respiratory function (forced vital capacity, FVC) and motor function evaluated with gait performance (Six\u2010Minute Walk Test, 6MWT) were assessed before the switch, and one and 2\u2009years after. Individual changes were compared using paired\u2010sample tests.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Forty\u2010seven adult patients were included. A stabilization of motor decline was observed: prior to switching, the 6MWT decreased by \u221227\u2009m\/year, whereas an improvement of +17\u2009m\/year was seen during the first year after the switch (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.001), followed by overall stability in the second year (\u221210\u2009m\/year,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.280). Respiratory changes were not statistically significant: a decline of 60\u2009mL\/year before the switch versus 10\u2009mL\/year after 1\u2009year (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.161), and 20\u2009mL\/year during the second year (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.346). Three patients died during follow\u2010up, with causes unrelated to the disease or treatment.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Gait deterioration halted during the first year after transitioning to avalglucosidase, with sustained stabilization thereafter, while respiratory parameters showed minimal change. For patients experiencing significant walking decline under alglucosidase alfa therapy, switching to avalglucosidase alfa resulted in disease stabilization, beginning with mild improvement in the first year and a return to pre\u2010switch baseline thereafter.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ene.70680","type":"journal-article","created":{"date-parts":[[2026,7,16]],"date-time":"2026-07-16T18:22:12Z","timestamp":1784226132000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Real\u2010Life Effectiveness After Switching to Avalglucosidase Alfa in Late\u2010Onset Pompe Disease Patients Worsening on Alglucosidase Alfa Therapy: A French Cohort Study"],"prefix":"10.1111","volume":"33","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":"Sabrina","family":"Sacconi","sequence":"additional","affiliation":[{"name":"Centre Hospitalier Universitaire de Nice, Syst\u00e8me Nerveux P\u00e9riph\u00e9rique and Muscle, H\u00f4pital Pasteur 2 Universit\u00e9 C\u00f4te D'azur  Nice France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nadjib","family":"Taouagh","sequence":"additional","affiliation":[{"name":"Centre de R\u00e9f\u00e9rence Des Maladies NeuroMusculaires Nord \u2013 Est \u2013 Ile\u2010de\u2010France, Filnemus  Garches France"},{"name":"Neurology Department APHP, Raymond Poincar\u00e9 University Hospital, FHU PHENIX  Garches 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