{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T21:43:24Z","timestamp":1791582204980,"version":"4.3.4"},"reference-count":33,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2023,4,17]],"date-time":"2023-04-17T00:00:00Z","timestamp":1681689600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100009480","name":"Stichting Vrienden van het Sophia","doi-asserted-by":"publisher","award":["W.OK18\u201013"],"award-info":[{"award-number":["W.OK18\u201013"]}],"id":[{"id":"10.13039\/501100009480","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2023,7]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Exercise has proven to be an effective adjuvant treatment to enzyme replacement therapy (ERT) in mildly affected adult Pompe patients. The aim of this study was to investigate the effects of a 12\u2010week tailored lifestyle intervention, consisting of physical training and a high protein diet (2 grams\/kg), in children with Pompe disease. This randomized controlled semi\u2010crossover trial investigated the effects of a lifestyle intervention on the primary outcome: exercise capacity. Secondary outcomes were: muscle strength, core stability, motor function, physical activity levels, quality of life, fatigue, fear of exercise, caloric intake, energy balance, body composition, and safety. Fourteen Pompe patients with a median age of 10.6 [IQR: 7.2\u201314.5], of whom six classic infantile patients, participated in the lifestyle intervention. At baseline, patients had a lower exercise capacity compared to healthy peers (median 70.3% [IQR: 54.8%\u201398.6%] of predicted). After the intervention, absolute Peak VO\n                    <jats:sub>2<\/jats:sub>\n                    improved significantly (1279\u2009mL\/min [1012.5\u20132006] vs. 1352\u2009mL\/min [1101.5\u20132069],\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.039), but not compared to the control period. Muscle strength of the hip flexors, hip abductors, elbow extensors, neck extensors, knee extensors, and core stability improved significantly compared to the control period. Children reported a significant increase on the change in health domain of quality of life, parents reported significantly better scores on the quality of life domains: physical functioning, change in health, family cohesion, and fatigue. A 12\u2010week tailored lifestyle intervention for children with Pompe disease seemed safe and led to improvements in muscle strength, core stability, quality of life, and parent\u2010reported fatigue. Pompe patients with a stable disease trajectory seemed to benefit the most from the intervention.\n                  <\/jats:p>","DOI":"10.1002\/jimd.12607","type":"journal-article","created":{"date-parts":[[2023,4,1]],"date-time":"2023-04-01T09:53:56Z","timestamp":1680342836000},"page":"605-617","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Physical training and high\u2010protein diet improved muscle strength, parent\u2010reported fatigue, and physical quality of life in children with Pompe disease"],"prefix":"10.1002","volume":"46","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9187-7892","authenticated-orcid":false,"given":"L. E.","family":"Scheffers","sequence":"first","affiliation":[{"name":"Department of pediatrics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center  Rotterdam The Netherlands"},{"name":"Respiratory Medicine and Allergology, Department of Pediatrics Erasmus MC University Medical Center\u2014Sophia Children's Hospital  Rotterdam The Netherlands"},{"name":"Department of Pediatric Gastroenterology Erasmus MC University Medical Center\u2014Sophia Children's Hospital  Rotterdam The Netherlands"},{"name":"Department of Pediatric Cardiology Erasmus MC University Medical Center\u2014Sophia Children's Hospital  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"O. C.","family":"Somers","sequence":"additional","affiliation":[{"name":"Department of pediatrics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"K.","family":"Dulfer","sequence":"additional","affiliation":[{"name":"Intensive Care and Department of Pediatric Surgery Erasmus MC University Medical Center\u2014Sophia Children's Hospital  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"G. C.","family":"Dieleman","sequence":"additional","affiliation":[{"name":"Department of Child and Adolescent Psychiatry\/Psychology Erasmus MC University Medical Center\u2014Sophia Children's Hospital  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S.","family":"Walet","sequence":"additional","affiliation":[{"name":"Division of Dietetics, Department of Internal Medicine Erasmus MC University Medical Center  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"L. J.","family":"van der Giessen","sequence":"additional","affiliation":[{"name":"Department of Pediatric Physiotherapy Erasmus MC University Medical Center  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A. T.","family":"van der Ploeg","sequence":"additional","affiliation":[{"name":"Department of pediatrics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. M. P.","family":"van den Hout","sequence":"additional","affiliation":[{"name":"Department of pediatrics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"L. E.","family":"van den Berg","sequence":"additional","affiliation":[{"name":"Department of pediatrics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center  Rotterdam The Netherlands"},{"name":"Department of Orthopedics and Sports Medicine Erasmus MC University Medical Center  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the exercise team","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,4,17]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2021.791701"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)61555-X"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545\u2010018\u20100166\u20103"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01482-w"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa0909859"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-018-0824-4"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-015-0303-0"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.20665"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2011.05.013"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2022.02.002"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01416-6"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.33.1.34"},{"key":"e_1_2_11_14_1","unstructured":"Center P.Secondary.2021www.pompevariantdatabase.nl"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12268"},{"key":"e_1_2_11_16_1","unstructured":"Castor.The randomization algorithm in castor. 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