{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T08:13:14Z","timestamp":1777882394906,"version":"3.51.4"},"reference-count":20,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"DOI":"10.13039\/100015362","name":"Amicus Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100015362","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Therapeutic Advances in Rare Disease"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Late-onset Pompe disease (LOPD) is caused by a deficiency of the acid \u03b1-glucosidase enzyme. In LOPD treatment, enzyme replacement therapy is delivered via intravenous infusion, typically in clinical settings. Cipaglucosidase alfa is delivered with the oral enzyme stabilizer miglustat (cipa\u2009+\u2009mig).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives:<\/jats:title>\n                    <jats:p>Evaluate the safety of cipa\u2009+\u2009mig home infusions.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design:<\/jats:title>\n                    <jats:p>Post hoc analysis of pooled safety data from three clinical trials in adults with LOPD (NCT02675465, NCT03729362, NCT04138277).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>The frequency and severity of infusion-associated reactions (IARs) during cipa\u2009+\u2009mig home and clinic administration were analyzed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>In total, 65\/151 patients (43.0%) received \u2a7e1 cipa\u2009+\u2009mig home treatment. Of 9185 treatments, 2024 (22.0%) were administered at home. IAR frequency was similar for home (1.3%, 26\/2024) and clinic (1.8%, 129\/7161). The most frequent IAR following home infusion was pyrexia (6.2% of patients). Two patients with \u2a7e1 home-based treatment experienced serious IARs.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Analyses support the safety of home cipa\u2009+\u2009mig treatment in eligible adults with LOPD.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/26330040261416943","type":"journal-article","created":{"date-parts":[[2026,1,31]],"date-time":"2026-01-31T12:36:26Z","timestamp":1769862986000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials"],"prefix":"10.1177","volume":"7","author":[{"given":"Henning","family":"Andersen","sequence":"first","affiliation":[{"name":"Aarhus Universitetshospital, Neurologisk Klinik, Kontorgangen, Indgang J, Plan 5, Kryspunkt 504, Palle Juul-Jensens Boulevard, Aarhus N 8200, Denmark"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jordi","family":"D\u00edaz-Manera","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne, UK"},{"name":"Neuromuscular Disorders Unit, Neurology Department, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain"},{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red en Enfermedades Raras, Barcelona, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ozlem","family":"Goker-Alpan","sequence":"additional","affiliation":[{"name":"Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tahseen","family":"Mozaffar","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of California, Irvine, CA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sheela","family":"Sitaraman Das","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brian","family":"Fox","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Farah","family":"Amon","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kinesha","family":"O\u2019Brien-Prince","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mitchell","family":"Goldman","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fred","family":"Holdbrook","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vipul","family":"Jain","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Barry J.","family":"Byrne","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2026,1,31]]},"reference":[{"key":"e_1_3_4_2_2","doi-asserted-by":"publisher","DOI":"10.1097\/01.gim.0000218152.87434.f3"},{"key":"e_1_3_4_3_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.22329"},{"key":"e_1_3_4_4_2","doi-asserted-by":"publisher","DOI":"10.1042\/bj0860011"},{"key":"e_1_3_4_5_2","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.125358"},{"key":"e_1_3_4_6_2","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2024.1451512"},{"key":"e_1_3_4_7_2","unstructured":"European Medicines Agency. Pombiliti: EPAR product information https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/pombiliti#ema-inpage-item-product-info (2023 accessed January 2025)."},{"key":"e_1_3_4_8_2","unstructured":"European Medicines Agency. Myozyme: summary of product characteristics https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/myozyme-epar-product-information_en.pdf (2011 accessed January 2025)."},{"key":"e_1_3_4_9_2","unstructured":"European Medicines Agency. Nexviadyme: summary of product characteristics https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/nexviadyme-epar-product-information_en.pdf (2022 accessed January 2025)."},{"key":"e_1_3_4_10_2","doi-asserted-by":"publisher","DOI":"10.1186\/s12883-017-0983-2"},{"key":"e_1_3_4_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2022.07.150"},{"key":"e_1_3_4_12_2","first-page":"100971","article-title":"The patients\u2019 perspective on home-based infusion: a longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy","volume":"35","author":"Heinrich R","year":"2023","unstructured":"Heinrich R, Claus F, Schoenfelder T. The patients\u2019 perspective on home-based infusion: a longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy. Mol Genet Metab Rep 2023; 35: 100971.","journal-title":"Mol Genet Metab Rep"},{"key":"e_1_3_4_13_2","doi-asserted-by":"publisher","DOI":"10.3390\/healthcare11081176"},{"key":"e_1_3_4_14_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-023-02919-8"},{"key":"e_1_3_4_15_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-023-02715-4"},{"key":"e_1_3_4_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00331-8"},{"key":"e_1_3_4_17_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-023-12096-0"},{"key":"e_1_3_4_18_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-024-12236-0"},{"key":"e_1_3_4_19_2","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-007-0686-8"},{"key":"e_1_3_4_20_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2024.108608"},{"key":"e_1_3_4_21_2","unstructured":"Electronic Medicines Compendium. Manual for patients with Pompe disease who receive home infusion of POMBILITI (cipaglucosidase alfa) https:\/\/www.medicines.org.uk\/emc\/rmm\/2804\/Document (2023 accessed August 2025)."}],"container-title":["Therapeutic Advances in Rare Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/26330040261416943","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/26330040261416943","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/26330040261416943","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T01:10:54Z","timestamp":1777597854000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/26330040261416943"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1]]},"references-count":20,"alternative-id":["10.1177\/26330040261416943"],"URL":"https:\/\/doi.org\/10.1177\/26330040261416943","relation":{},"ISSN":["2633-0040","2633-0040"],"issn-type":[{"value":"2633-0040","type":"print"},{"value":"2633-0040","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,1]]},"article-number":"26330040261416943"}}