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Treatments include enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), but the effects are limited depending on the age of initiation and clinical phenotype. Thus, this study aims to assess the effects of treatments on MPS IVA patients compared to untreated MPS IVA patients and an age\u2010matched control group.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We used activity of daily living (ADL) survey with 4\u00a0sections: \u201cmovement,\u201d \u201cmovement with cognition,\u201d \u201ccognition,\u201d and \u201cother MPS symptoms.\u201d Lower scores indicate more assistance required. This study included 161 patients, 270 total surveys, and 70 patients with longitudinal data.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We describe 134\u00a0severe patients and 25 attenuated patients. ERT and HSCT treatment improved only the \u201cother MPS symptoms\u201d section in severe patients. There were no differences between ERT and HSCT severe patient scores. A 19\u2010year\u2010old male patient, who had robust physical training, provided a significant increase in \u201cmovement\u201d without treatment, suggesting the importance of exercise.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Overall, this ADL questionnaire has demonstrated validation and reliability in assessing the MPS IVA patients and therapeutic efficacy.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mgg3.1806","type":"journal-article","created":{"date-parts":[[2021,10,10]],"date-time":"2021-10-10T08:55:23Z","timestamp":1633856123000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy"],"prefix":"10.1002","volume":"9","author":[{"given":"Hui","family":"Chen","sequence":"first","affiliation":[{"name":"University of Delaware Newark DE USA"},{"name":"Nemours\/Alfred I. duPont Hospital for 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