{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T07:50:06Z","timestamp":1793346606816,"version":"4.5.0"},"reference-count":38,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2021,11,17]],"date-time":"2021-11-17T00:00:00Z","timestamp":1637107200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2021,11,17]],"date-time":"2021-11-17T00:00:00Z","timestamp":1637107200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100000024","name":"Canadian Institutes of Health Research","doi-asserted-by":"publisher","award":["171684"],"award-info":[{"award-number":["171684"]}],"id":[{"id":"10.13039\/501100000024","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Trials"],"published-print":{"date-parts":[[2021,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec>\n                <jats:title>Background<\/jats:title>\n                <jats:p>Mucopolysaccharidoses (MPS) are a group of inherited metabolic diseases characterized by chronic, progressive multi-system manifestations with varying degrees of severity. Disease-modifying therapies exist to treat some types of MPS; however, they are not curative, underscoring the need to identify and evaluate co-interventions that optimize functioning, participation in preferred activities, and quality of life. A Canadian pediatric MPS registry is under development and may serve as a platform to launch randomized controlled trials to evaluate such interventions. To promote the standardized collection of patient\/family-reported and clinical outcomes considered important to patients\/families, health care providers (HCPs), and policymakers, the choice of outcomes to include in the registry will be informed by a core outcome set (COS). We aim to establish a patient-oriented COS for pediatric MPS using a multi-stakeholder approach.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>In step 1 of the six-step process to develop the COS, we will identify relevant outcomes through a rapid literature review and candidate outcomes survey. A two-phase screening approach will be implemented to identify eligible publications, followed by extraction of outcomes and other pre-specified data elements. Simultaneously, we will conduct a candidate outcomes survey with children with MPS and their families to identify outcomes most important to them. In step 2, HCPs experienced in treating patients with MPS will be invited to review the list of outcomes generated in step 1 and identify additional clinically relevant outcomes. We will then ask patients\/families, HCPs, and policymakers to rate the outcomes in a set of Delphi Surveys (step 3), and to participate in a subsequent consensus meeting to finalize the COS (step 4). Step 5 involves establishing a set of outcome measurement instruments for the COS. Finally, we will disseminate the COS to knowledge users (step 6).<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Discussion<\/jats:title>\n                <jats:p>The proposed COS will inform the choice of outcomes to include in the MPS registry and, more broadly, promote the standardized collection of patient-oriented outcomes for pediatric MPS research. By involving patients\/families from the earliest stage of the research, we will ensure that the COS will be relevant to those who will ultimately benefit from the research.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Trial registration<\/jats:title>\n                <jats:p>PROSPERO <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/www.comet-initiative.org\/Studies\/Details\/1924\">CRD42021267531<\/jats:ext-link>, COMET<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s13063-021-05791-8","type":"journal-article","created":{"date-parts":[[2021,11,19]],"date-time":"2021-11-19T11:21:43Z","timestamp":1637320903000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Establishing a core outcome set for mucopolysaccharidoses (MPS) in children: study protocol for a rapid literature review, candidate outcomes survey, and Delphi surveys"],"prefix":"10.1186","volume":"22","author":[{"given":"Alison H.","family":"Howie","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kylie","family":"Tingley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michal","family":"Inbar-Feigenberg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John J.","family":"Mitchell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy J.","family":"Butcher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Offringa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maureen","family":"Smith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kim","family":"Angel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenifer","family":"Gentle","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra","family":"Wyatt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe M.","family":"Campeau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alicia","family":"Chan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pranesh","family":"Chakraborty","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Farah","family":"El Turk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eva","family":"Mamak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aizeddin","family":"Mhanni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Becky","family":"Skidmore","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca","family":"Sparkes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvia","family":"Stockler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8374-6542","authenticated-orcid":false,"given":"Beth K.","family":"Potter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"in collaboration with the INFORM RARE Network","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,11,17]]},"reference":[{"key":"5791_CR1","volume-title":"The Mucopolysaccharidoses","author":"EF Neufeld","year":"2006","unstructured":"Neufeld EF, Muenzer J. The Mucopolysaccharidoses. Valle D, Beaudet AL, Vogelstein B, Kinzler KW, Antonarakis SE, Ballabio A, editors. Scriver\u2019s online metabolic and molecular bases of inherited diseases: In; 2006."},{"issue":"3","key":"5791_CR2","doi-asserted-by":"publisher","first-page":"227","DOI":"10.1016\/j.ymgme.2017.05.016","volume":"121","author":"SA Khan","year":"2017","unstructured":"Khan SA, Peracha H, Ballhausen D, Wiesbauer A, Rohrbach M, Gautschi M, et al. Epidemiology of mucopolysaccharidoses. Mol Genet Metab. 2017;121(3):227\u201340.","journal-title":"Mol Genet Metab."},{"issue":"5 SUPPL","key":"5791_CR3","doi-asserted-by":"publisher","first-page":"27","DOI":"10.1016\/j.jpeds.2004.01.052","volume":"144","author":"J Muenzer","year":"2004","unstructured":"Muenzer J. The mucopolysaccharidoses: a heterogeneous group of disorders with variable pediatric presentations. J Pediatr. 2004;144(5 SUPPL):27\u201334.","journal-title":"J Pediatr."},{"key":"5791_CR4","doi-asserted-by":"crossref","unstructured":"Barone R, Pellico A, Pittal\u00e0 A, Gasperini S. Neurobehavioral phenotypes of neuronopathic mucopolysaccharidoses. Ital J Pediatr. 2018;44(S2).","DOI":"10.1186\/s13052-018-0561-2"},{"issue":"2","key":"5791_CR5","doi-asserted-by":"publisher","first-page":"127","DOI":"10.1038\/s10038-018-0534-8","volume":"64","author":"H Kobayashi","year":"2019","unstructured":"Kobayashi H. Recent trends in mucopolysaccharidosis research. J Hum Genet [Internet]. 2019;64(2):127\u201337 Available from: https:\/\/doi.org\/10.1038\/s10038-018-0534-8.","journal-title":"J Hum Genet [Internet]."},{"issue":"SUPPL 5","key":"5791_CR6","doi-asserted-by":"publisher","first-page":"49","DOI":"10.1093\/rheumatology\/ker396","volume":"50","author":"V Valayannopoulos","year":"2011","unstructured":"Valayannopoulos V, Wijburg FA. Therapy for the mucopolysaccharidoses. Rheumatology. 2011;50(SUPPL 5):49\u201359.","journal-title":"Rheumatology."},{"key":"5791_CR7","doi-asserted-by":"publisher","first-page":"778","DOI":"10.1001\/jamapediatrics.2015.0754","volume":"169","author":"HJ Vernon","year":"2015","unstructured":"Vernon HJ. Inborn errors of metabolism: advances in diagnosis and therapy. JAMA Pediatr. 2015;169:778\u201382.","journal-title":"JAMA Pediatr."},{"key":"5791_CR8","unstructured":"World Health Organization. International classification of functioning, disability and health\u00a0: children & youth version\u00a0: ICF-CY. 2007."},{"issue":"8","key":"5791_CR9","doi-asserted-by":"publisher","first-page":"637","DOI":"10.1016\/0021-9681(67)90041-0","volume":"20","author":"D Schwartz","year":"1967","unstructured":"Schwartz D, Lellouch J. Explanatory and pragmatic attitudes in therapeutical trials. J Chronic Dis. 1967;20(8):637\u201348.","journal-title":"J Chronic Dis."},{"issue":"2","key":"5791_CR10","doi-asserted-by":"publisher","first-page":"217","DOI":"10.31887\/DCNS.2011.13.2\/npatsopoulos","volume":"13","author":"NA Patsopoulos","year":"2011","unstructured":"Patsopoulos NA. A pragmatic view on pragmatic trials. Dialogues Clin Neurosci. 2011;13(2):217\u201324.","journal-title":"Dialogues Clin Neurosci."},{"issue":"1","key":"5791_CR11","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13063-015-1023-4","volume":"16","author":"T Kennedy-Martin","year":"2015","unstructured":"Kennedy-Martin T, Curtis S, Faries D, Robinson S, Johnston J. A literature review on the representativeness of randomized controlled trial samples and implications for the external validity of trial results. Trials. 2015;16(1):1\u201314.","journal-title":"Trials."},{"key":"5791_CR12","doi-asserted-by":"crossref","unstructured":"Loudon K, Treweek S, Sullivan F, Donnan P, Thorpe KE, Zwarenstein M. The PRECIS-2 tool: designing trials that are fit for purpose. BMJ. 2015;350.","DOI":"10.1136\/bmj.h2147"},{"key":"5791_CR13","doi-asserted-by":"publisher","unstructured":"Li G, Sajobi TT, Menon BK, Korngut L, Lowerison M, James M, et al. Registry-based randomized controlled trials- what are the advantages, challenges, and areas for future research? J Clin Epidemiol [Internet]. 2016;80:16\u201324. Available from: https:\/\/doi.org\/10.1016\/j.jclinepi.2016.08.003","DOI":"10.1016\/j.jclinepi.2016.08.003"},{"issue":"1","key":"5791_CR14","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13063-017-2394-5","volume":"19","author":"KA Mc Cord","year":"2018","unstructured":"Mc Cord KA, Al-Shahi Salman R, Treweek S, Gardner H, Strech D, Whiteley W, et al. Routinely collected data for randomized trials: promises, barriers, and implications. Trials. 2018;19(1):1\u20139.","journal-title":"Trials."},{"key":"5791_CR15","doi-asserted-by":"publisher","unstructured":"Mathes T, Buehn S, Prengel P, Pieper D. Registry-based randomized controlled trials merged the strength of randomized controlled trails and observational studies and give rise to more pragmatic trials. J Clin Epidemiol [Internet]. 2018;93:120\u20137. Available from: https:\/\/doi.org\/10.1016\/j.jclinepi.2017.09.017","DOI":"10.1016\/j.jclinepi.2017.09.017"},{"issue":"17","key":"5791_CR16","doi-asserted-by":"publisher","first-page":"1579","DOI":"10.1056\/NEJMp1310102","volume":"369","author":"M Lauer","year":"2013","unstructured":"Lauer M, D\u2019Agostino R. The randomized registry trial \u2014 the next disruptive technology in clinical research? N Engl J Med. 2013;369(17):1579\u201381.","journal-title":"N Engl J Med."},{"issue":"3","key":"5791_CR17","doi-asserted-by":"publisher","first-page":"163","DOI":"10.1136\/archdischild-2016-312117","volume":"103","author":"J Webbe","year":"2018","unstructured":"Webbe J, Sinha I, Gale C. Core Outcome Sets. Arch Dis Child Educ Pract Ed. 2018;103(3):163\u20136.","journal-title":"Arch Dis Child Educ Pract Ed."},{"issue":"S1","key":"5791_CR18","doi-asserted-by":"publisher","first-page":"2011","DOI":"10.1186\/1745-6215-12-S1-A70","volume":"12","author":"PR Williamson","year":"2011","unstructured":"Williamson PR, Altman DG, Blazeby JM, Clarke M, Gargon E. The COMET (Core Outcome Measures in Effectiveness Trials) initiative. Trials. 2011;12(S1):2011.","journal-title":"Trials."},{"key":"5791_CR19","doi-asserted-by":"crossref","unstructured":"Pugliese, Michael; Tingley, Kylie; Chow, Andrea; Pallone, Nicole; Smith, Maureen; Chakraborty, Pranesh; Potter BK. Core outcome sets for pediatric medium chain acyl-coA dehydrogenase deficiency and phenylketonuria. Pediatrics.\u00a02021;148(2):1\u201313..","DOI":"10.1542\/peds.2020-037747"},{"issue":"1","key":"5791_CR20","doi-asserted-by":"publisher","first-page":"F76","DOI":"10.1136\/archdischild-2017-314560","volume":"104","author":"BSR Allin","year":"2019","unstructured":"Allin BSR, Hall NJ, Ross AR, Marven SS, Kurinczuk JJ, Knight M. Development of a gastroschisis core outcome set. Arch Dis Child Fetal Neonatal Ed. 2019;104(1):F76\u201382.","journal-title":"Arch Dis Child Fetal Neonatal Ed."},{"issue":"6","key":"5791_CR21","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1371\/journal.pone.0129514","volume":"10","author":"NL Harman","year":"2015","unstructured":"Harman NL, Bruce IA, Kirkham JJ, Tierney S, Callery P, O\u2019Brien K, et al. The importance of integration of stakeholder views in core outcome set development: Otitis Media with Effusion in children with cleft palate. PLoS One. 2015;10(6):1\u201322.","journal-title":"PLoS One."},{"key":"5791_CR22","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1590\/2326-4594-iem-18-0016","volume":"7","author":"A Metryka","year":"2019","unstructured":"Metryka A, Brown N, Mercer J, Wilkinson S, Jones S, Williamson P, et al. Toward a core outcome set for head, neck, and respiratory disease in mucopolysaccharidosis type II: systematic literature review and assessment of heterogeneity in outcome reporting. J Inborn Errors Metab Screen. 2019;7:1\u201312.","journal-title":"J Inborn Errors Metab Screen."},{"issue":"1","key":"5791_CR23","doi-asserted-by":"publisher","first-page":"1","DOI":"10.5582\/irdr.2020.01011","volume":"9","author":"J Zhou","year":"2020","unstructured":"Zhou J, Lin J, Leung WT, Wang L. A basic understanding of mucopolysaccharidosis: incidence, clinical features, diagnosis, and management. Intractable Rare Dis Res. 2020;9(1):1\u20139.","journal-title":"Intractable Rare Dis Res."},{"issue":"1","key":"5791_CR24","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13063-019-3230-x","volume":"20","author":"JJ Kirkham","year":"2019","unstructured":"Kirkham JJ, Gorst S, Altman DG, Blazeby JM, Clarke M, Tunis S, et al. Core Outcome Set-STAndardised Protocol Items: the COS-STAP Statement. Trials. 2019;20(1):1\u20137.","journal-title":"Trials."},{"issue":"1","key":"5791_CR25","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13063-017-2327-3","volume":"18","author":"BK Potter","year":"2017","unstructured":"Potter BK, Hutton B, Clifford TJ, Pallone N, Smith M, Stockler S, et al. Establishing core outcome sets for phenylketonuria (PKU) and medium-chain Acyl-CoA dehydrogenase (MCAD) deficiency in children: study protocol for systematic reviews and Delphi surveys. Trials. 2017;18(1):1\u201310.","journal-title":"Trials."},{"issue":"11","key":"5791_CR26","doi-asserted-by":"publisher","first-page":"1297","DOI":"10.1016\/j.jaac.2020.07.905","volume":"59","author":"S Monga","year":"2020","unstructured":"Monga S, Monsour A, Stallwood E, Desai R, Cleverley K, Courtney D, et al. Core outcome set development for adolescent major depressive disorder clinical trials: a registered report. J Am Acad Child Adolesc Psychiatry [Internet]. 2020;59(11):1297\u20138 Available from: https:\/\/doi.org\/10.1016\/j.jaac.2020.07.905.","journal-title":"J Am Acad Child Adolesc Psychiatry [Internet]."},{"issue":"Suppl 3","key":"5791_CR27","first-page":"1","volume":"18","author":"PR Williamson","year":"2017","unstructured":"Williamson PR, Altman DG, Bagley H, Barnes KL, Blazeby JM, Brookes ST, et al. The COMET Handbook: Version 1.0. Trials. 2017;18(Suppl 3):1\u201350.","journal-title":"Trials."},{"key":"5791_CR28","doi-asserted-by":"publisher","unstructured":"Hamel C, Michaud A, Thuku M, Skidmore B, Stevens A, Nussbaumer-Streit B, et al. Defining rapid reviews: a systematic scoping review and thematic analysis of definitions and defining characteristics of rapid reviews. J Clin Epidemiol [Internet]. 2021;129:74\u201385 Available from: https:\/\/doi.org\/10.1016\/j.jclinepi.2020.09.041.","DOI":"10.1016\/j.jclinepi.2020.09.041"},{"key":"5791_CR29","doi-asserted-by":"publisher","first-page":"40","DOI":"10.1016\/j.jclinepi.2016.01.021","volume":"75","author":"J McGowan","year":"2016","unstructured":"McGowan J, Sampson M, Salzwedel DM, Cogo E, Foerster V, Lefebvre C. PRESS peer review of electronic search strategies: 2015 guideline\u00a0statement. J Clin Epidemiol [Internet]. 2016;75:40\u20136 Available from: https:\/\/doi.org\/10.1016\/j.jclinepi.2016.01.021.","journal-title":"J Clin Epidemiol [Internet]."},{"key":"5791_CR30","volume-title":"Introduction to the methods guide for medical test reviews","author":"D Matchar","year":"2012","unstructured":"Matchar D. Introduction to the methods guide for medical test reviews. In: Methods Guide for Medical Test Reviews. AHRQ Publi; 2012."},{"issue":"1","key":"5791_CR31","doi-asserted-by":"publisher","first-page":"e20153967","DOI":"10.1542\/peds.2015-3967","volume":"138","author":"MZ Kapadia","year":"2016","unstructured":"Kapadia MZ, Joachim KC, Balasingham C, Cohen E, Mahant S, Nelson K, et al. A core outcome set for children with feeding tubes and neurologic impairment: a systematic review. Pediatrics. 2016;138(1):e20153967.","journal-title":"Pediatrics"},{"issue":"7","key":"5791_CR32","doi-asserted-by":"publisher","first-page":"745","DOI":"10.1016\/j.jclinepi.2013.11.013","volume":"67","author":"M Boers","year":"2014","unstructured":"Boers M, Kirwan JR, Wells G, Beaton D, Gossec L, D\u2019Agostino MA, et al. Developing core outcome measurement sets for clinical trials: OMERACT filter 2.0. J Clin Epidemiol. 2014;67(7):745\u201353.","journal-title":"J Clin Epidemiol."},{"key":"5791_CR33","unstructured":"GRADE [Internet]. [cited 2021 Aug 4]. Available from: https:\/\/www.gradeworkinggroup.org\/"},{"issue":"1","key":"5791_CR34","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13063-015-1128-9","volume":"17","author":"CAC Prinsen","year":"2016","unstructured":"Prinsen CAC, Vohra S, Rose MR, Boers M, Tugwell P, Clarke M, et al. How to select outcome measurement instruments for outcomes included in a \u201cCore Outcome Set\u201d - a practical guideline. Trials [Internet]. 2016;17(1):1\u201310 Available from: https:\/\/doi.org\/10.1186\/s13063-016-1555-2.","journal-title":"Trials [Internet]."},{"issue":"1","key":"5791_CR35","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/1745-6215-16-1","volume":"16","author":"A Moza","year":"2015","unstructured":"Moza A, Benstoem C, Autschbach R, Stoppe C, Goetzenich A. A core outcome set for all types of cardiac surgery effectiveness trials: a study protocol for an international eDelphi survey to achieve consensus on what to measure and the subsequent selection of measurement instruments. Trials [Internet]. 2015;16(1):1\u20137 Available from: https:\/\/doi.org\/10.1186\/s13063-015-1072-8.","journal-title":"Trials [Internet]."},{"issue":"9","key":"5791_CR36","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1136\/bmjopen-2017-016371","volume":"7","author":"L Rose","year":"2017","unstructured":"Rose L, Agar M, Burry LD, Campbell N, Clarke M, Lee J, et al. Development of core outcome sets for effectiveness trials of interventions to prevent and\/or treat delirium (Del-COrS): study protocol. BMJ Open. 2017;7(9):1\u20139.","journal-title":"BMJ Open."},{"issue":"1","key":"5791_CR37","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/1750-1172-6-72","volume":"6","author":"M Scarpa","year":"2011","unstructured":"Scarpa M, Alm\u00e1ssy Z, Beck M, Bodamer O, Bruce IA, De Meirleir L, et al. Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease. Orphanet J Rare Dis. 2011;6(1):1\u201318.","journal-title":"Orphanet J Rare Dis."},{"issue":"1\u20132","key":"5791_CR38","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1016\/j.ymgme.2020.08.007","volume":"131","author":"JH van der Lee","year":"2020","unstructured":"van der Lee JH, Morton J, Adams HR, Clarke L, Eisengart JB, Escolar ML, et al. Therapy development for the mucopolysaccharidoses: updated consensus recommendations for neuropsychological endpoints. Mol Genet Metab. 2020;131(1\u20132):181\u201396.","journal-title":"Mol Genet Metab."}],"updated-by":[{"DOI":"10.1186\/s13063-021-05893-3","type":"correction","label":"Correction","source":"publisher","updated":{"date-parts":[[2021,12,9]],"date-time":"2021-12-09T00:00:00Z","timestamp":1639008000000}}],"container-title":["Trials"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s13063-021-05791-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1186\/s13063-021-05791-8\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s13063-021-05791-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,12,9]],"date-time":"2021-12-09T11:14:38Z","timestamp":1639048478000},"score":1,"resource":{"primary":{"URL":"https:\/\/trialsjournal.biomedcentral.com\/articles\/10.1186\/s13063-021-05791-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,11,17]]},"references-count":38,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2021,12]]}},"alternative-id":["5791"],"URL":"https:\/\/doi.org\/10.1186\/s13063-021-05791-8","relation":{"correction":[{"id-type":"doi","id":"10.1186\/s13063-021-05893-3","asserted-by":"object"}]},"ISSN":["1745-6215"],"issn-type":[{"value":"1745-6215","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,11,17]]},"assertion":[{"value":"25 August 2021","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"3 November 2021","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"17 November 2021","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"9 December 2021","order":4,"name":"change_date","label":"Change Date","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Correction","order":5,"name":"change_type","label":"Change Type","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"A Correction to this paper has been published:","order":6,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"https:\/\/doi.org\/10.1186\/s13063-021-05893-3","URL":"https:\/\/doi.org\/10.1186\/s13063-021-05893-3","order":7,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Ethics approval has been granted for steps 1a and 1b by the University of Ottawa Research Ethics Board. Approval of the Delphi surveys will be sought from the University of Ottawa Research Ethics Board.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable (no individual person\u2019s data are included in this manuscript).","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"The authors declare that they have no competing interests.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"816"}}