{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T22:57:23Z","timestamp":1793055443612,"version":"4.5.0"},"reference-count":70,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2023,6,16]],"date-time":"2023-06-16T00:00:00Z","timestamp":1686873600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2023,6,16]],"date-time":"2023-06-16T00:00:00Z","timestamp":1686873600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"DOI":"10.13039\/501100001826","name":"ZonMw","doi-asserted-by":"publisher","award":["09150161910230"],"award-info":[{"award-number":["09150161910230"]}],"id":[{"id":"10.13039\/501100001826","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"name":"TKI Life Sciences and Health"},{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["BioDrugs"],"published-print":{"date-parts":[[2023,9]]},"DOI":"10.1007\/s40259-023-00609-2","type":"journal-article","created":{"date-parts":[[2023,6,16]],"date-time":"2023-06-16T09:02:46Z","timestamp":1686906166000},"page":"685-698","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions"],"prefix":"10.1007","volume":"37","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2377-3108","authenticated-orcid":false,"given":"Imke A. M.","family":"Ditters","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9098-1356","authenticated-orcid":false,"given":"Harmke A.","family":"van Kooten","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9161-3301","authenticated-orcid":false,"given":"Nadine A. M. E.","family":"van der Beek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacqueline F.","family":"Hardon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gamida","family":"Ismailova","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1452-2219","authenticated-orcid":false,"given":"Esther","family":"Brusse","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle E.","family":"Kruijshaar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3359-1324","authenticated-orcid":false,"given":"Ans T.","family":"van der Ploeg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8091-263X","authenticated-orcid":false,"given":"Johanna M. P.","family":"van den Hout","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1276-0931","authenticated-orcid":false,"given":"Hidde H.","family":"Huidekoper","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2023,6,16]]},"reference":[{"issue":"15","key":"609_CR1","doi-asserted-by":"publisher","first-page":"1396","DOI":"10.1056\/NEJMoa0909859","volume":"362","author":"AT van der Ploeg","year":"2010","unstructured":"van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, et al. A randomized study of alglucosidase alfa in late-onset Pompe\u2019s disease. N Engl J Med. 2010;362(15):1396\u2013406.","journal-title":"N Engl J Med"},{"issue":"9646","key":"609_CR2","doi-asserted-by":"publisher","first-page":"1342","DOI":"10.1016\/S0140-6736(08)61555-X","volume":"372","author":"AT van der Ploeg","year":"2008","unstructured":"van der Ploeg AT, Reuser AJ. Pompe\u2019s disease. Lancet. 2008;372(9646):1342\u201353.","journal-title":"Lancet"},{"issue":"19","key":"609_CR3","doi-asserted-by":"publisher","first-page":"e1756","DOI":"10.1212\/WNL.0000000000008441","volume":"93","author":"L Harlaar","year":"2019","unstructured":"Harlaar L, Hogrel J-Y, Perniconi B, Kruijshaar ME, Rizopoulos D, Taouagh N, et al. Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease. Neurology. 2019;93(19):e1756\u201367.","journal-title":"Neurology"},{"issue":"2","key":"609_CR4","doi-asserted-by":"publisher","first-page":"253","DOI":"10.1007\/s10545-015-9889-6","volume":"39","author":"D G\u00fcng\u00f6r","year":"2016","unstructured":"G\u00fcng\u00f6r D, Kruijshaar ME, Plug I, Rizopoulos D, Kanters TA, Wens SC, et al. Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up. J Inherit Metab Dis. 2016;39(2):253\u201360.","journal-title":"J Inherit Metab Dis"},{"issue":"11","key":"609_CR5","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0242630","volume":"15","author":"C Perraudin","year":"2020","unstructured":"Perraudin C, Bourdin A, Vicino A, Kuntzer T, Bugnon O, Berger J. Home-based subcutaneous immunoglobulin for chronic inflammatory demyelinating polyneuropathy patients: a Swiss cost-minimization analysis. PLoS\u00a0One. 2020;15(11): e0242630.","journal-title":"PLoS\u00a0One"},{"key":"609_CR6","unstructured":"FDA. Highlights of prescribing information MYOZYME\u00ae (alglucosidase alfa) injectable for intravenous infusion: FDA [updated 05-2019]. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2014\/125141s219lbl.pdf."},{"key":"609_CR7","unstructured":"Myozyme: EPAR\u2014Product information: European Medicines Agency; 2022 [updated 25-08-2022]. https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/myozyme-epar-product-information_en.pdf."},{"issue":"8","key":"609_CR8","doi-asserted-by":"publisher","first-page":"2943","DOI":"10.1007\/s00415-021-10475-z","volume":"268","author":"S Wenninger","year":"2021","unstructured":"Wenninger S, Gutschmidt K, Wirner C, Einvag K, Montagnese F, Schoser B. The impact of interrupting enzyme replacement therapy in late-onset Pompe disease. J Neurol. 2021;268(8):2943\u201350.","journal-title":"J Neurol"},{"issue":"4","key":"609_CR9","doi-asserted-by":"publisher","first-page":"1181","DOI":"10.1111\/ene.15222","volume":"29","author":"C Tard","year":"2022","unstructured":"Tard C, Salort-Campana E, Michaud M, Spinazzi M, NadajPakleza A, Durr H, et al. Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID-19 pandemic. Eur J Neurol. 2022;29(4):1181\u20136.","journal-title":"Eur J Neurol"},{"issue":"9","key":"609_CR10","doi-asserted-by":"publisher","first-page":"2716","DOI":"10.3390\/jcm9092716","volume":"9","author":"A Fiumara","year":"2020","unstructured":"Fiumara A, Lanzafame G, Arena A, Sapuppo A, Raudino F, Pratic\u00f2 A, et al. COVID-19 pandemic outbreak and its psychological impact on patients with rare lysosomal diseases. J Clin Med. 2020;9(9):2716.","journal-title":"J Clin Med"},{"issue":"3","key":"609_CR11","doi-asserted-by":"publisher","first-page":"170","DOI":"10.1016\/j.ymgme.2020.04.002","volume":"130","author":"A Sechi","year":"2020","unstructured":"Sechi A, Macor D, Valent S, Da Riol RM, Zanatta M, Spinelli A, et al. Impact of COVID-19 related healthcare crisis on treatments for patients with lysosomal storage disorders, the first Italian experience. Mol Genet Metab. 2020;130(3):170\u20131.","journal-title":"Mol Genet Metab"},{"issue":"1","key":"609_CR12","doi-asserted-by":"publisher","first-page":"341","DOI":"10.1186\/s13023-020-01619-x","volume":"15","author":"C Lampe","year":"2020","unstructured":"Lampe C, Dionisi-Vici C, Bellettato CM, Paneghetti L, van Lingen C, Bond S, et al. The impact of COVID-19 on rare metabolic patients and healthcare providers: results from two MetabERN surveys. Orphanet J Rare Dis. 2020;15(1):341.","journal-title":"Orphanet J Rare Dis"},{"issue":"22","key":"609_CR13","doi-asserted-by":"publisher","first-page":"959","DOI":"10.1212\/WNL.0000000000009566","volume":"94","author":"AC Guidon","year":"2020","unstructured":"Guidon AC, Amato AA. COVID-19 and neuromuscular disorders. Neurology. 2020;94(22):959\u201369.","journal-title":"Neurology"},{"issue":"10","key":"609_CR14","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pmed.1001885","volume":"12","author":"EI Benchimol","year":"2015","unstructured":"Benchimol EI, Smeeth L, Guttmann A, Harron K, Moher D, Petersen I, et al. The REporting of studies Conducted using Observational Routinely-collected health Data (RECORD) statement. PLoS Med. 2015;12(10): e1001885.","journal-title":"PLoS Med"},{"issue":"3","key":"609_CR15","doi-asserted-by":"publisher","first-page":"456","DOI":"10.1016\/j.ymgme.2012.09.015","volume":"107","author":"AT van der Ploeg","year":"2012","unstructured":"van der Ploeg AT, Barohn R, Carlson L, Charrow J, Clemens PR, Hopkin RJ, et al. Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa. Mol Genet Metab. 2012;107(3):456\u201361.","journal-title":"Mol Genet Metab"},{"key":"609_CR16","unstructured":"National Institutes of Health NCI. Common Terminology Criteria for Adverse Events, version 5.0: National Institutes of Health, National Cancer Institute; 2017. https:\/\/ctep.cancer.gov\/protocoldevelopment\/electronic_applications\/docs\/CTCAE_v5_Quick_Reference_5x7.pdf [updated Nov 27]."},{"key":"609_CR17","unstructured":"Central Committee on Research Involving Human Subjects. L4. SAEs. https:\/\/english.ccmo.nl\/investigators\/standard-research-file\/l-safety-information\/l4-saes."},{"key":"609_CR18","unstructured":"Council for International Organizations of Medical Sciences (CIOMS). Guidelines for preparing core clinical-safety information on drugs: Including new proposals for investigator\u2019s brochures. Switzerland: Council for International Organizations of Medical Sciences; 1999."},{"key":"609_CR19","doi-asserted-by":"publisher","first-page":"90","DOI":"10.1038\/gim.2016.70","volume":"19","author":"JM de Vries","year":"2017","unstructured":"de Vries JM, Kuperus E, Hoogeveen-Westerveld M, Kroos MA, Wens SCA, Stok M, et al. Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy. Genet Med. 2017;19:90.","journal-title":"Genet Med"},{"issue":"4","key":"609_CR20","doi-asserted-by":"publisher","first-page":"1107","DOI":"10.1182\/blood.V82.4.1107.1107","volume":"82","author":"A Zimran","year":"1993","unstructured":"Zimran A, Hollak CE, Abrahamov A, van Oers MH, Kelly M, Beutler E. Home treatment with intravenous enzyme replacement therapy for Gaucher disease: an international collaborative study of 33 patients. Blood. 1993;82(4):1107\u20139.","journal-title":"Blood"},{"issue":"2","key":"609_CR21","doi-asserted-by":"publisher","first-page":"355","DOI":"10.1093\/ndt\/gfi221","volume":"21","author":"GE Linthorst","year":"2006","unstructured":"Linthorst GE, Vedder AC, Ormel EE, Aerts JM, Hollak CE. Home treatment for Fabry disease: practice guidelines based on 3 years experience in The Netherlands. Nephrol Dial Transplant. 2006;21(2):355\u201360.","journal-title":"Nephrol Dial Transplant"},{"issue":"6","key":"609_CR22","doi-asserted-by":"publisher","first-page":"984","DOI":"10.1007\/s10545-007-0686-8","volume":"30","author":"J Cox-Brinkman","year":"2007","unstructured":"Cox-Brinkman J, Timmermans RG, Wijburg FA, Donker WE, van de Ploeg AT, Aerts JM, et al. Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe. J Inherit Metab Dis. 2007;30(6):984.","journal-title":"J Inherit Metab Dis"},{"issue":"2\u20133","key":"609_CR23","doi-asserted-by":"publisher","first-page":"123","DOI":"10.1016\/j.ymgme.2010.06.011","volume":"101","author":"BK Burton","year":"2010","unstructured":"Burton BK, Guffon N, Roberts J, van der Ploeg AT, Jones SA, HOS Investigators. Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II\u2014data from the Hunter Outcome Survey. Mol Genet Metab. 2010;101(2\u20133):123\u20139.","journal-title":"Mol Genet Metab"},{"issue":"6","key":"609_CR24","doi-asserted-by":"publisher","first-page":"733","DOI":"10.1007\/s10545-008-0980-0","volume":"31","author":"S Bagewadi","year":"2008","unstructured":"Bagewadi S, Roberts J, Mercer J, Jones S, Stephenson J, Wraith JE. Home treatment with Elaprase and Naglazyme is safe in patients with mucopolysaccharidoses types II and VI, respectively. J Inherit Metab Dis. 2008;31(6):733\u20137.","journal-title":"J Inherit Metab Dis"},{"key":"609_CR25","unstructured":"Joep HJ, Kamphoven LD, Kroos MA, van der Ploeg AT, Duncker DJ, Reuser AJJ. Both low and high uptake forms of acid a-glucosidase target to muscle of KO mice with Pompe's disease. In: Center EMUM, editor. Pompe's disease; the mouse model as model in the development of enzyme therapy; 2004. p. 90\u2013104. https:\/\/repub.eur.nl\/pub\/15246\/Kamphoven_Joep%20H%20J_18%20feb%202004.pdf."},{"issue":"Pt 3","key":"609_CR26","doi-asserted-by":"publisher","first-page":"619","DOI":"10.1042\/BJ20050364","volume":"389","author":"Y Zhu","year":"2005","unstructured":"Zhu Y, Li X, McVie-Wylie A, Jiang C, Thurberg BL, Raben N, et al. Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice. Biochem J. 2005;389(Pt 3):619\u201328.","journal-title":"Biochem J"},{"issue":"6","key":"609_CR27","doi-asserted-by":"publisher","first-page":"743","DOI":"10.1002\/mus.10381","volume":"27","author":"LPF Winkel","year":"2003","unstructured":"Winkel LPF, Kamphoven JHJ, van den Hout HJMP, Severijnen LA, van Doorn PA, Reuser AJJ, et al. Morphological changes in muscle tissue of patients with infantile Pompe\u2019s disease receiving enzyme replacement therapy. Muscle Nerve. 2003;27(6):743\u201351.","journal-title":"Muscle Nerve"},{"issue":"5","key":"609_CR28","doi-asserted-by":"publisher","first-page":"581","DOI":"10.1016\/j.jpeds.2004.01.046","volume":"144","author":"JE Wraith","year":"2004","unstructured":"Wraith JE, Clarke LA, Beck M, Kolodny EH, Pastores GM, Muenzer J, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-l-iduronidase (laronidase). J Pediatr. 2004;144(5):581\u20138.","journal-title":"J Pediatr"},{"issue":"3","key":"609_CR29","doi-asserted-by":"publisher","first-page":"795","DOI":"10.1007\/s10545-011-9285-9","volume":"34","author":"XS Zhang","year":"2011","unstructured":"Zhang XS, Brondyk W, Lydon JT, Thurberg BL, Piepenhagen PA. Biotherapeutic target or sink: analysis of the macrophage mannose receptor tissue distribution in murine models of lysosomal storage diseases. J Inherit Metab Dis. 2011;34(3):795\u2013809.","journal-title":"J Inherit Metab Dis"},{"issue":"13","key":"609_CR30","doi-asserted-by":"publisher","first-page":"291","DOI":"10.21037\/atm.2019.04.15","volume":"7","author":"HV Do","year":"2019","unstructured":"Do HV, Khanna R, Gotschall R. Challenges in treating Pompe disease: an industry perspective. Ann Transl Med. 2019;7(13):291.","journal-title":"Ann Transl Med."},{"issue":"4","key":"609_CR31","doi-asserted-by":"publisher","first-page":"1123","DOI":"10.1016\/j.jaci.2021.02.012","volume":"147","author":"A Cianferoni","year":"2021","unstructured":"Cianferoni A. Non-IgE-mediated anaphylaxis. J Allergy Clin Immunol. 2021;147(4):1123\u201331.","journal-title":"J Allergy Clin Immunol"},{"issue":"11","key":"609_CR32","doi-asserted-by":"publisher","first-page":"843","DOI":"10.2165\/00002018-200124110-00005","volume":"24","author":"KL Drain","year":"2001","unstructured":"Drain KL, Volcheck GW. Preventing and managing drug-induced anaphylaxis. Drug Saf. 2001;24(11):843\u201353.","journal-title":"Drug Saf"},{"issue":"12","key":"609_CR33","doi-asserted-by":"publisher","first-page":"1355","DOI":"10.1111\/j.1365-2222.1996.tb00536.x","volume":"26","author":"MM van der Klauw","year":"1996","unstructured":"van der Klauw MM, Wilson JH, Stricker BH. Drug-associated anaphylaxis: 20 years of reporting in The Netherlands (1974\u20131994) and review of the literature. Clin Exp Allergy. 1996;26(12):1355\u201363.","journal-title":"Clin Exp Allergy"},{"issue":"7","key":"609_CR34","doi-asserted-by":"publisher","first-page":"41","DOI":"10.1007\/s11882-021-01018-7","volume":"21","author":"SR Joshi","year":"2021","unstructured":"Joshi SR, Khan DA. Non-IgE-mediated drug hypersensitivity reactions. Curr Allergy Asthma Rep. 2021;21(7):41.","journal-title":"Curr Allergy Asthma Rep"},{"issue":"5","key":"609_CR35","doi-asserted-by":"publisher","first-page":"898","DOI":"10.1038\/s41436-019-0738-0","volume":"22","author":"AA Khan","year":"2020","unstructured":"Khan AA, Case LE, Herbert M, DeArmey S, Jones H, Crisp K, et al. Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature. Genet Med. 2020;22(5):898\u2013907. https:\/\/doi.org\/10.1038\/s41436-019-0738-0.","journal-title":"Genet Med"},{"issue":"4","key":"609_CR36","doi-asserted-by":"publisher","first-page":"499","DOI":"10.1007\/s10545-016-9917-1","volume":"39","author":"A Broomfield","year":"2016","unstructured":"Broomfield A, Jones SA, Hughes SM, Bigger BW. The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders. J Inherit Metab Dis. 2016;39(4):499\u2013512.","journal-title":"J Inherit Metab Dis"},{"issue":"1","key":"609_CR37","doi-asserted-by":"publisher","first-page":"26","DOI":"10.1016\/j.ymgme.2009.08.003","volume":"99","author":"PS Kishnani","year":"2010","unstructured":"Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, et al. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010;99(1):26\u201333.","journal-title":"Mol Genet Metab"},{"issue":"8","key":"609_CR38","doi-asserted-by":"publisher","first-page":"729","DOI":"10.1097\/GIM.0b013e3182174703","volume":"13","author":"SG Banugaria","year":"2011","unstructured":"Banugaria SG, Prater SN, Ng YK, Kobori JA, Finkel RS, Ladda RL, et al. The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease. Genet Med. 2011;13(8):729\u201336.","journal-title":"Genet Med"},{"issue":"2","key":"609_CR39","doi-asserted-by":"publisher","first-page":"305","DOI":"10.1007\/s10545-014-9707-6","volume":"38","author":"CM van Gelder","year":"2015","unstructured":"van Gelder CM, Hoogeveen-Westerveld M, Kroos MA, Plug I, van der Ploeg AT, Reuser AJJ. Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease. J Inherit Metab Dis. 2015;38(2):305\u201314.","journal-title":"J Inherit Metab Dis"},{"issue":"3","key":"609_CR40","doi-asserted-by":"publisher","first-page":"383","DOI":"10.1007\/s10545-015-9912-y","volume":"39","author":"CM van Gelder","year":"2016","unstructured":"van Gelder CM, Poelman E, Plug I, Hoogeveen-Westerveld M, van der Beek N, Reuser AJJ, et al. Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study. J Inherit Metab Dis. 2016;39(3):383\u201390.","journal-title":"J Inherit Metab Dis"},{"issue":"3","key":"609_CR41","doi-asserted-by":"publisher","first-page":"143","DOI":"10.1177\/1756285609103324","volume":"2","author":"C Angelini","year":"2009","unstructured":"Angelini C, Semplicini C, Tonin P, Filosto M, Pegoraro E, Sorar\u00f9 G, et al. Progress in enzyme replacement therapy in glycogen storage disease type II. Ther Adv Neurol Disord. 2009;2(3):143\u201353.","journal-title":"Ther Adv Neurol Disord"},{"issue":"1","key":"609_CR42","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-018-29170-7","volume":"8","author":"S Figueroa-Bonaparte","year":"2018","unstructured":"Figueroa-Bonaparte S, Llauger J, Segovia S, Belmonte I, Pedrosa I, Montiel E, et al. Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study. Sci Rep. 2018;8(1): 10898.","journal-title":"Sci Rep"},{"issue":"23","key":"609_CR43","doi-asserted-by":"publisher","first-page":"2365","DOI":"10.1212\/WNL.0000000000004711","volume":"89","author":"E Kuperus","year":"2017","unstructured":"Kuperus E, Kruijshaar ME, Wens SCA, de Vries JM, Favejee MM, van der Meijden JC, et al. Long-term benefit of enzyme replacement therapy in Pompe disease: a 5-year prospective study. Neurology. 2017;89(23):2365\u201373.","journal-title":"Neurology"},{"issue":"1","key":"609_CR44","doi-asserted-by":"publisher","first-page":"159","DOI":"10.1007\/s00415-017-8686-6","volume":"265","author":"WN L\u00f6scher","year":"2018","unstructured":"L\u00f6scher WN, Huemer M, Stulnig TM, Simschitz P, Iglseder S, Eggers C, et al. Pompe disease in Austria: clinical, genetic and epidemiological aspects. J Neurol. 2018;265(1):159\u201364.","journal-title":"J Neurol"},{"issue":"10","key":"609_CR45","doi-asserted-by":"publisher","first-page":"999","DOI":"10.1002\/humu.20374","volume":"27","author":"AL Montalvo","year":"2006","unstructured":"Montalvo AL, Bembi B, Donnarumma M, Filocamo M, Parenti G, Rossi M, et al. Mutation profile of the GAA gene in 40 Italian patients with late onset glycogen storage disease type II. Hum Mutat. 2006;27(10):999\u20131006.","journal-title":"Hum Mutat"},{"issue":"4","key":"609_CR46","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1016\/j.ymgme.2017.10.008","volume":"122","author":"M Mori","year":"2017","unstructured":"Mori M, Haskell G, Kazi Z, Zhu X, DeArmey SM, Goldstein JL, et al. Sensitivity of whole exome sequencing in detecting infantile- and late-onset Pompe disease. Mol Genet Metab. 2017;122(4):189\u201397.","journal-title":"Mol Genet Metab"},{"issue":"1","key":"609_CR47","doi-asserted-by":"publisher","first-page":"41","DOI":"10.1016\/j.ymgme.2010.09.002","volume":"102","author":"GK Papadimas","year":"2011","unstructured":"Papadimas GK, Terzis G, Methenitis S, Spengos K, Papadopoulos C, Vassilopoulou S, et al. Body composition analysis in late-onset Pompe disease. Mol Genet Metab. 2011;102(1):41\u20133.","journal-title":"Mol Genet Metab"},{"issue":"5","key":"609_CR48","doi-asserted-by":"publisher","first-page":"837","DOI":"10.1007\/s10545-012-9451-8","volume":"35","author":"C Regnery","year":"2012","unstructured":"Regnery C, Kornblum C, Hanisch F, Vielhaber S, Strigl-Pill N, Grunert B, et al. 36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy. J Inherit Metab Dis. 2012;35(5):837\u201345.","journal-title":"J Inherit Metab Dis"},{"issue":"12","key":"609_CR49","doi-asserted-by":"publisher","first-page":"2783","DOI":"10.1007\/s00415-018-9065-7","volume":"265","author":"O Scheidegger","year":"2018","unstructured":"Scheidegger O, Leupold D, Sauter R, Findling O, R\u00f6sler KM, Hundsberger T. 36-Months follow-up assessment after cessation and resuming of enzyme replacement therapy in late onset Pompe disease: data from the Swiss Pompe Registry. J Neurol. 2018;265(12):2783\u20138.","journal-title":"J Neurol"},{"issue":"6","key":"609_CR50","doi-asserted-by":"publisher","first-page":"937","DOI":"10.1007\/s10545-018-0243-7","volume":"41","author":"C Semplicini","year":"2018","unstructured":"Semplicini C, Letard P, De Antonio M, Taouagh N, Perniconi B, Bouhour F, et al. Late-onset Pompe disease in France: molecular features and epidemiology from a nationwide study. J Inherit Metab Dis. 2018;41(6):937\u201346.","journal-title":"J Inherit Metab Dis"},{"issue":"6","key":"609_CR51","doi-asserted-by":"publisher","first-page":"1205","DOI":"10.1007\/s10545-018-0166-3","volume":"41","author":"JC van der Meijden","year":"2018","unstructured":"van der Meijden JC, Kruijshaar ME, Harlaar L, Rizopoulos D, van der Beek N, van der Ploeg AT. Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy. J Inherit Metab Dis. 2018;41(6):1205\u201314.","journal-title":"J Inherit Metab Dis"},{"issue":"4","key":"609_CR52","doi-asserted-by":"publisher","first-page":"465","DOI":"10.1016\/j.pjnns.2018.05.002","volume":"52","author":"G Witkowski","year":"2018","unstructured":"Witkowski G, Konopko M, Rola R, Lugowska A, Ryglewicz D, Sienkiewicz-Jarosz H. Enzymatic replacement therapy in patients with late-onset Pompe disease\u20146-year follow up. Neurol Neurochir Pol. 2018;52(4):465\u20139.","journal-title":"Neurol Neurochir Pol"},{"issue":"2","key":"609_CR53","doi-asserted-by":"publisher","first-page":"110","DOI":"10.1212\/01.wnl.0000252798.25690.76","volume":"68","author":"MA Kroos","year":"2007","unstructured":"Kroos MA, Pomponio RJ, Hagemans ML, Keulemans JL, Phipps M, DeRiso M, et al. Broad spectrum of Pompe disease in patients with the same c.-32-13T->G haplotype. Neurology. 2007;68(2):110\u20135.","journal-title":"Neurology"},{"key":"609_CR54","doi-asserted-by":"publisher","first-page":"90","DOI":"10.1016\/j.omtn.2017.03.001","volume":"7","author":"E van der Wal","year":"2017","unstructured":"van der Wal E, Bergsma AJ, Pijnenburg JM, van der Ploeg AT, Pijnappel W. Antisense oligonucleotides promote exon inclusion and correct the common c.-32-13T>G GAA splicing variant in Pompe disease. Mol Ther Nucleic Acids. 2017;7:90\u2013100.","journal-title":"Mol Ther Nucleic Acids"},{"key":"609_CR55","doi-asserted-by":"publisher","DOI":"10.1038\/srep36182","volume":"6","author":"E Masat","year":"2016","unstructured":"Masat E, Lafor\u00eat P, De Antonio M, Corre G, Perniconi B, Taouagh N, et al. Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients. Sci Rep. 2016;6: 36182.","journal-title":"Sci Rep"},{"issue":"3","key":"609_CR56","doi-asserted-by":"publisher","first-page":"319","DOI":"10.1016\/j.ymgme.2009.07.001","volume":"98","author":"SE Lipinski","year":"2009","unstructured":"Lipinski SE, Lipinski MJ, Burnette A, Platts-Mills TA, Wilson WG. Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to alglucosidase alfa. Mol Genet Metab. 2009;98(3):319\u201321.","journal-title":"Mol Genet Metab"},{"issue":"3","key":"609_CR57","doi-asserted-by":"publisher","first-page":"198","DOI":"10.1159\/000446154","volume":"169","author":"M Capanoglu","year":"2016","unstructured":"Capanoglu M, DibekMisirlioglu E, Azkur D, Vezir E, Guvenir H, Gunduz M, et al. IgE-mediated hypersensitivity and desensitisation with recombinant enzymes in Pompe disease and type I and type VI mucopolysaccharidosis. Int Arch Allergy Immunol. 2016;169(3):198\u2013202.","journal-title":"Int Arch Allergy Immunol"},{"issue":"11","key":"609_CR58","doi-asserted-by":"publisher","first-page":"801","DOI":"10.1016\/j.nmd.2016.07.006","volume":"26","author":"L Gallay","year":"2016","unstructured":"Gallay L, Petiot P, Durieu I, Streichenberger N, Berard F. SWORD: a simplified desensitization protocol for enzyme replacement therapy in adult Pompe disease. Neuromuscul Disord. 2016;26(11):801\u20134.","journal-title":"Neuromuscul Disord"},{"issue":"3","key":"609_CR59","doi-asserted-by":"publisher","first-page":"329","DOI":"10.1203\/PDR.0b013e3181b24e94","volume":"66","author":"PS Kishnani","year":"2009","unstructured":"Kishnani PS, Corzo D, Leslie ND, Gruskin D, Van der Ploeg A, Clancy JP, et al. Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease. Pediatr Res. 2009;66(3):329\u201335.","journal-title":"Pediatr Res"},{"issue":"5","key":"609_CR60","doi-asserted-by":"publisher","first-page":"e448","DOI":"10.1542\/peds.113.5.e448","volume":"113","author":"JMP Van den Hout","year":"2004","unstructured":"Van den Hout JMP, Kamphoven JHJ, Winkel LPF, Arts WFM, De Klerk JBC, Loonen MCB, et al. Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk. Pediatrics. 2004;113(5):e448\u201357.","journal-title":"Pediatrics"},{"issue":"6","key":"609_CR61","doi-asserted-by":"publisher","first-page":"628","DOI":"10.1097\/MOP.0b013e3282f161f2","volume":"19","author":"TA Burrow","year":"2007","unstructured":"Burrow TA, Hopkin RJ, Leslie ND, Tinkle BT, Grabowski GA. Enzyme reconstitution\/replacement therapy for lysosomal storage diseases. Curr Opin Pediatr. 2007;19(6):628\u201335.","journal-title":"Curr Opin Pediatr"},{"key":"609_CR62","doi-asserted-by":"publisher","first-page":"63","DOI":"10.1007\/8904_2016_8","volume":"34","author":"Z Karimian","year":"2017","unstructured":"Karimian Z, Whitley CB, Rudser KD, Utz JRJ. Delayed infusion reactions to enzyme replacement therapies. JIMD Rep. 2017;34:63\u201370.","journal-title":"JIMD Rep"},{"issue":"6","key":"609_CR63","doi-asserted-by":"publisher","first-page":"330","DOI":"10.12968\/bjon.2006.15.6.20681","volume":"15","author":"A Milligan","year":"2006","unstructured":"Milligan A, Hughes D, Goodwin S, Richfield L, Mehta A. Intravenous enzyme replacement therapy: better in home or hospital? Br J Nurs. 2006;15(6):330\u20133.","journal-title":"Br J Nurs"},{"key":"609_CR64","doi-asserted-by":"publisher","first-page":"85","DOI":"10.1016\/j.ymgmr.2017.06.005","volume":"12","author":"D Concolino","year":"2017","unstructured":"Concolino D, Amico L, Cappellini MD, Cassinerio E, Conti M, Donati MA, et al. Home infusion program with enzyme replacement therapy for Fabry disease: the experience of a large Italian collaborative group. Mol Genet Metab Rep. 2017;12:85\u201391.","journal-title":"Mol Genet Metab Rep"},{"issue":"3","key":"609_CR65","doi-asserted-by":"publisher","first-page":"234","DOI":"10.1016\/j.ymgme.2009.04.007","volume":"97","author":"BK Burton","year":"2009","unstructured":"Burton BK, Wiesman C, Paras A, Kim K, Katz R. Home infusion therapy is safe and enhances compliance in patients with mucopolysaccharidoses. Mol Genet Metab. 2009;97(3):234\u20136.","journal-title":"Mol Genet Metab"},{"issue":"4","key":"609_CR66","doi-asserted-by":"publisher","first-page":"415","DOI":"10.1016\/j.bcmd.2015.09.002","volume":"55","author":"D Elstein","year":"2015","unstructured":"Elstein D, Abrahamov A, Oz A, Arbel N, Baris H, Zimran A. 13,845 home therapy infusions with velaglucerase alfa exemplify safety of velaglucerase alfa and increased compliance to every-other-week intravenous enzyme replacement therapy for Gaucher disease. Blood Cells Mol Dis. 2015;55(4):415\u20138.","journal-title":"Blood Cells Mol Dis"},{"issue":"10","key":"609_CR67","first-page":"1002","volume":"24","author":"L Nalysnyk","year":"2018","unstructured":"Nalysnyk L, Sugarman R, Cele C, Uyei J, Ward A. Budget impact analysis of eliglustat for the treatment of gaucher disease type 1 in the United States. J Manag Care Spec Pharm. 2018;24(10):1002\u20138.","journal-title":"J Manag Care Spec Pharm"},{"issue":"12","key":"609_CR68","doi-asserted-by":"publisher","first-page":"1104","DOI":"10.1111\/j.1365-2362.2010.02363.x","volume":"40","author":"JF Guest","year":"2010","unstructured":"Guest JF, Jenssen T, Houge G, Aaseboe W, T\u00f8ndel C, Svarstad E. Modelling the resource implications of managing adults with Fabry disease in Norway favours home infusion. Eur J Clin Investig. 2010;40(12):1104\u201312.","journal-title":"Eur J Clin Investig"},{"issue":"1","key":"609_CR69","doi-asserted-by":"publisher","first-page":"179","DOI":"10.1186\/s13023-017-0731-0","volume":"12","author":"TA Kanters","year":"2017","unstructured":"Kanters TA, van der Ploeg AT, Kruijshaar ME, Rizopoulos D, Redekop WK, Rutten-van M\u04e7lken M, et al. Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease. Orphanet J Rare Dis. 2017;12(1):179.","journal-title":"Orphanet J Rare Dis"},{"issue":"1\u20132","key":"609_CR70","doi-asserted-by":"publisher","first-page":"118","DOI":"10.1016\/j.ymgme.2011.07.004","volume":"104","author":"AH El-Gharbawy","year":"2011","unstructured":"El-Gharbawy AH, Mackey J, DeArmey S, Westby G, Grinnell SG, Malovrh P, et al. An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions. Mol Genet Metab. 2011;104(1\u20132):118\u201322.","journal-title":"Mol Genet Metab"}],"container-title":["BioDrugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40259-023-00609-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40259-023-00609-2\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40259-023-00609-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,16]],"date-time":"2023-08-16T15:14:37Z","timestamp":1692198877000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40259-023-00609-2"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,6,16]]},"references-count":70,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2023,9]]}},"alternative-id":["609"],"URL":"https:\/\/doi.org\/10.1007\/s40259-023-00609-2","relation":{},"ISSN":["1173-8804","1179-190X"],"issn-type":[{"value":"1173-8804","type":"print"},{"value":"1179-190X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,6,16]]},"assertion":[{"value":"21 May 2023","order":1,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"16 June 2023","order":2,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"This study was partly supported by ZonMw (project number 09150161910230), Prinses Beatrix Spierfonds, TKI Life Sciences and Health, and Sanofi.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Funding"}},{"value":"Imke A. M. Ditters, Harmke A. van Kooten, Jacqueline F. Hardon, Gamida Ismailova, Esther Brusse, Michelle E. Kruijshaar and Hidde H. Huidekoper declare no conflicts of interest. Ans T. van der Ploeg received funding for research, clinical trials, and advisory fees from Sanofi, Amicus Therapeutics, Spark Therapeutics, Denali Therapeutics and Takeda working on ERT or next\u00adgeneration therapies in the field of Pompe disease, other lysosomal storage diseases or neuromuscular disorders, under agreements with Erasmus MC University Medical Center and the relevant industry. Nadine A. M. E. van der Beek received funding for research, clinical trials, and advisory fees from Sanofi working on ERT or next\u00adgeneration therapies in the field of Pompe disease, other lysosomal storage diseases or neuromuscular disorders, under an agreement between this company and Erasmus MC University Medical Center and the relevant industry. She also received a Veni grant from ZonMW (project no. 09150161910230). Johanna M. P. van den Hout received funding for research, clinical trials, and advisory fees from Sanofi, Denali Therapeutics and Takeda working on ERT or next\u00adgeneration therapies in the field of Pompe disease, other lysosomal storage diseases or neuromuscular disorders, under agreements with Erasmus MC University Medical Center and the relevant industry.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"Not applicable.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"We received consent to participate from all participants and\/or their caregivers.","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}},{"value":"We received consent for publication from all participants and\/or their caregivers.","order":6,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"De\u00adidentified data will be shared upon reasonable request to the corresponding author from any qualified investigator for the sole purpose of replicating procedures and results presented in this article, in agreement with EU legislation on the general data protection regulation.","order":7,"name":"Ethics","group":{"name":"EthicsHeading","label":"Data availability"}},{"value":"Not applicable.","order":8,"name":"Ethics","group":{"name":"EthicsHeading","label":"Code availability"}},{"value":"Conceptualization: IAM Ditters, NAME van der Beek, ME Kruijshaar, AT van der Ploeg, JMP van den Hout\nand HH Huidekoper. Data curation: IAM Ditters. Formal analysis: IAM Ditters, JMP van den Hout and HH\nHuidekoper. Investigation: IAM Ditters, JMP van den Hout and HH Huidekoper. Methodology: IAM\nDitters, ME Kruijshaar, AT van der Ploeg, JMP van den Hout and HH Huidekoper. Project administration:\nIAM Ditters, JMP van den Hout and HH Huidekoper. Resources: IAM Ditters, HA van Kooten, NAME van\nder Beek, JF Hardon, G Ismailova, E Brusse, AT van der Ploeg, JMP van den Hout and HH Huidekoper.\nSupervision: AT van der Ploeg, JMP van den Hout and HH Huidekoper. Validation: IAM Ditters, JMP van\nden Hout and HH Huidekoper. Visualization: IAM Ditters, AT van der Ploeg, JMP van den Hout and HH\nHuidekoper. Writing original draft: IAM Ditters, AT van der Ploeg, JMP van den Hout and HH Huidekoper.\nWriting \u2013 review & editing: IAM Ditters, HA van Kooten, NAME van der Beek, JF Hardon, G Ismailova, E\nBrusse, ME Kruijshaar, AT van der Ploeg, JMP van den Hout and HH Huidekoper. All authors read and\napproved the final manuscript.","order":9,"name":"Ethics","group":{"name":"EthicsHeading","label":"Author contributions"}}]}}