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. 2021 Dec 4;16:501. doi: 10.1186/s13023-021-02118-3

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© The Author(s) 2021

Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.

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Fig. 1 — Study design. ^aIn addition to the assessments performed at baseline shown in the figure, the screening period includes: obtaining/evaluating informed consent/assent, inclusion/exclusion criteria, genotyping, demographic information and medical history. ^bHeight and weight were measured at baseline and at months 12 and 24 only. BSID-III Bayley Scales of Infant Development, third edition; DAS-II Differential Ability Scales, second edition; EQ-5D five-dimension EuroQol questionnaire; HRQoL health-related quality of life; HS-FOCUS Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale; VABS-II Vineland Adaptive Behavior Scales, second edition