{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,5]],"date-time":"2026-08-05T07:46:00Z","timestamp":1785915960710,"version":"3.56.0"},"reference-count":36,"publisher":"Elsevier BV","issue":"8","license":[{"start":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T00:00:00Z","timestamp":1785542400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T00:00:00Z","timestamp":1785542400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82203515"],"award-info":[{"award-number":["82203515"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100017696","name":"Guangzhou Medical University Guangzhou Women and Children&apos;s Medical Center","doi-asserted-by":"publisher","award":["2020BS011"],"award-info":[{"award-number":["2020BS011"]}],"id":[{"id":"10.13039\/100017696","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Transplantation and Cellular Therapy"],"published-print":{"date-parts":[[2026,8]]},"DOI":"10.1016\/j.jtct.2026.04.006","type":"journal-article","created":{"date-parts":[[2026,4,25]],"date-time":"2026-04-25T15:49:25Z","timestamp":1777132165000},"page":"970.e1-970.e10","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Long-Term Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Mucopolysaccharidosis IVA: A 10-Year Follow-Up from a Single-Center Experience in China"],"prefix":"10.1016","volume":"32","author":[{"ORCID":"https:\/\/orcid.org\/0009-0003-4728-6456","authenticated-orcid":false,"given":"Weiwei","family":"Gong","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hua","family":"Jiang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuhua","family":"Qu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"4","key":"10.1016\/j.jtct.2026.04.006_bib0001","doi-asserted-by":"crossref","first-page":"1517","DOI":"10.3390\/ijms21041517","article-title":"Mucopolysaccharidosis IVA: diagnosis, treatment, and management","volume":"21","author":"Sawamoto","year":"2020","journal-title":"Int J Mol Sci"},{"issue":"1","key":"10.1016\/j.jtct.2026.04.006_bib0002","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1186\/s13023-022-02219-7","article-title":"Addressing the need for patient-friendly medical communications: adaptation of the 2019 recommendations for the management of MPS VI and MPS IVA","volume":"17","author":"Bruce","year":"2022","journal-title":"Orphanet J Rare Dis"},{"issue":"5","key":"10.1016\/j.jtct.2026.04.006_bib0003","doi-asserted-by":"crossref","first-page":"1341","DOI":"10.1007\/s13346-020-00714-7","article-title":"Biodegradable polyethylene glycol hydrogels for sustained release and enhanced stability of rhGALNS enzyme","volume":"10","author":"Jain","year":"2020","journal-title":"Drug Deliv Transl Res"},{"issue":"1","key":"10.1016\/j.jtct.2026.04.006_bib0004","first-page":"104","article-title":"Enzyme replacement therapy interruption in mucopolysaccharidosis type IVA patients and its impact in different clinical outcomes","volume":"58","author":"Politei","year":"2021","journal-title":"JIMD Rep"},{"key":"10.1016\/j.jtct.2026.04.006_bib0005","first-page":"143","article-title":"Clinical utility of elosulfase alfa in the treatment of Morquio A syndrome","volume":"16","author":"Lee","year":"2022","journal-title":"Drug Des Devel Ther"},{"key":"10.1016\/j.jtct.2026.04.006_bib0006","article-title":"Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome)","volume":"42","author":"Sung","year":"2025","journal-title":"Mol Genet Metab Rep"},{"key":"10.1016\/j.jtct.2026.04.006_bib0007","article-title":"Clinical characteristics and effects of enzyme replacement therapy with elosulfase alfa in Korean patients with mucopolysaccharidosis type IVA","volume":"31","author":"Lee","year":"2022","journal-title":"Mol Genet Metab Rep"},{"issue":"11","key":"10.1016\/j.jtct.2026.04.006_bib0008","doi-asserted-by":"crossref","first-page":"1153","DOI":"10.1038\/s10038-019-0662-9","article-title":"Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future","volume":"64","author":"Chen","year":"2019","journal-title":"J Hum Genet"},{"issue":"5","key":"10.1016\/j.jtct.2026.04.006_bib0009","doi-asserted-by":"crossref","first-page":"515","DOI":"10.1007\/s11914-020-00620-4","article-title":"Bone-specific drug delivery for osteoporosis and rare skeletal disorders","volume":"18","author":"Sawamoto","year":"2020","journal-title":"Curr Osteoporos Rep"},{"issue":"Suppl 1","key":"10.1016\/j.jtct.2026.04.006_bib0010","doi-asserted-by":"crossref","first-page":"S10","DOI":"10.1016\/j.pedneo.2022.10.001","article-title":"Current and new therapies for mucopolysaccharidoses","volume":"64","author":"Penon-Portmann","year":"2023","journal-title":"Pediatr Neonatol"},{"issue":"4","key":"10.1016\/j.jtct.2026.04.006_bib0011","doi-asserted-by":"crossref","first-page":"443","DOI":"10.4103\/tjo.TJO-D-23-00137","article-title":"Mucopolysaccharidosis","volume":"13","author":"Bhakthaganesh","year":"2023","journal-title":"Taiwan J Ophthalmol"},{"key":"10.1016\/j.jtct.2026.04.006_bib0012","doi-asserted-by":"crossref","DOI":"10.1155\/2020\/2408402","article-title":"Mucopolysaccharidoses I and II: brief review of therapeutic options and supportive\/palliative therapies","volume":"2020","author":"Nan","year":"2020","journal-title":"Biomed Res Int"},{"issue":"2","key":"10.1016\/j.jtct.2026.04.006_bib0013","doi-asserted-by":"crossref","first-page":"189","DOI":"10.3390\/biom11020189","article-title":"Mucopolysaccharidosis type I: current treatments, limitations, and prospects for improvement","volume":"11","author":"Hampe","year":"2021","journal-title":"Biomolecules"},{"issue":"7","key":"10.1016\/j.jtct.2026.04.006_bib0014","doi-asserted-by":"crossref","first-page":"e226","DOI":"10.1016\/j.bbmt.2019.02.012","article-title":"Hematopoietic stem cell transplantation for mucopolysaccharidoses: past, present, and future","volume":"25","author":"Taylor","year":"2019","journal-title":"Biol Blood Marrow Transplant"},{"issue":"10","key":"10.1016\/j.jtct.2026.04.006_bib0015","doi-asserted-by":"crossref","first-page":"1103","DOI":"10.1007\/s40265-019-01147-4","article-title":"Therapeutic options for mucopolysaccharidoses: current and emerging treatments","volume":"79","author":"Sawamoto","year":"2019","journal-title":"Drugs"},{"issue":"10","key":"10.1016\/j.jtct.2026.04.006_bib0016","doi-asserted-by":"crossref","first-page":"1286","DOI":"10.1002\/ajmg.a.32281","article-title":"Growth charts for patients affected with Morquio A disease","volume":"146A","author":"Monta\u00f1o","year":"2008","journal-title":"Am J Med Genet A"},{"issue":"1","key":"10.1016\/j.jtct.2026.04.006_bib0017","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1038\/s41409-024-02439-4","article-title":"Hematopoietic stem cell transplantation in children with mucopolysaccharidosis IVA: single center experience","volume":"60","author":"Yalcin","year":"2025","journal-title":"Bone Marrow Transplant"},{"issue":"22","key":"10.1016\/j.jtct.2026.04.006_bib0018","doi-asserted-by":"crossref","DOI":"10.3390\/ijms242216148","article-title":"Mucopolysaccharidosis IVA: current disease models and drawbacks","volume":"24","author":"Leal","year":"2023","journal-title":"Int J Mol Sci"},{"issue":"3","key":"10.1016\/j.jtct.2026.04.006_bib0019","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1002\/jimd.12491","article-title":"Investigation of GALNS variants and genotype-phenotype correlations in a large cohort of patients with mucopolysaccharidosis type IVA","volume":"45","author":"Yi","year":"2022","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.jtct.2026.04.006_bib0020","doi-asserted-by":"crossref","first-page":"45","DOI":"10.2147\/TACG.S141881","article-title":"Determination of genotypic and clinical characteristics of Colombian patients with mucopolysaccharidosis IVA","volume":"11","author":"Tapiero-Rodriguez","year":"2018","journal-title":"Appl Clin Genet"},{"issue":"11","key":"10.1016\/j.jtct.2026.04.006_bib0021","doi-asserted-by":"crossref","first-page":"7073","DOI":"10.1007\/s11033-014-3383-3","article-title":"Computational analysis of human N-acetylgalactosamine-6-sulfate sulfatase enzyme: an update ingenotype-phenotype correlation for Morquio a","volume":"41","author":"Olarte-Avellaneda","year":"2014","journal-title":"Mol Biol Rep"},{"key":"10.1016\/j.jtct.2026.04.006_bib0022","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1007\/s10545-007-0529-7","article-title":"International Morquio A registry: clinical manifestation and natural course of Morquio A disease","volume":"30","author":"Monta\u00f1o","year":"2007","journal-title":"J Inherit Metab Dis"},{"issue":"2","key":"10.1016\/j.jtct.2026.04.006_bib0023","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1007\/s10545-012-9459-0","article-title":"Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA","volume":"36","author":"Hendriksz","year":"2013","journal-title":"J Inherit Metab Dis"},{"issue":"1","key":"10.1016\/j.jtct.2026.04.006_bib0024","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1186\/s13023-021-01761-0","article-title":"Morquio A syndrome and effect of enzyme replacement therapy in different age groups of Turkish patients: a case series","volume":"16","author":"K\u0131lavuz","year":"2021","journal-title":"Orphanet J Rare Dis"},{"issue":"5","key":"10.1016\/j.jtct.2026.04.006_bib0025","doi-asserted-by":"crossref","first-page":"971","DOI":"10.1007\/s00247-022-05585-3","article-title":"A pictorial review of the radiographic skeletal findings in Morquio syndrome (mucopolysaccharidosis type IV)","volume":"53","author":"Padash","year":"2023","journal-title":"Pediatr Radiol"},{"key":"10.1016\/j.jtct.2026.04.006_bib0026","doi-asserted-by":"crossref","first-page":"534","DOI":"10.1097\/BPO.0b013e318259fe57","article-title":"The lower extremity in Morquio syndrome","volume":"32","author":"Dhawale","year":"2012","journal-title":"J Pediatr Orthop"},{"key":"10.1016\/j.jtct.2026.04.006_bib0027","doi-asserted-by":"crossref","first-page":"931","DOI":"10.2174\/138920111795542615","article-title":"Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment","volume":"12","author":"Tomatsu","year":"2011","journal-title":"Curr Pharm Biotechnol"},{"issue":"2","key":"10.1016\/j.jtct.2026.04.006_bib0028","doi-asserted-by":"crossref","first-page":"108","DOI":"10.3390\/diagnostics10020108","article-title":"Long-term outcomes of early enzyme replacement therapy for mucopolysaccharidosis IV: clinical case studies of two siblings","volume":"10","author":"Barak","year":"2020","journal-title":"Diagnostics (Basel)"},{"key":"10.1016\/j.jtct.2026.04.006_bib0029","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1080\/21678707.2016.1214572","article-title":"Current therapies for Morquio A syndrome and their clinical outcomes","volume":"4","author":"Sawamoto","year":"2016","journal-title":"Expert Opin Orphan Drugs"},{"issue":"20","key":"10.1016\/j.jtct.2026.04.006_bib0030","doi-asserted-by":"crossref","DOI":"10.3390\/ijms232012693","article-title":"Sex difference leads to differential gene expression patterns and therapeutic efficacy in mucopolysaccharidosis IVA murine model receiving AAV8 gene therapy","volume":"23","author":"Piechnik","year":"2022","journal-title":"Int J Mol Sci"},{"issue":"5","key":"10.1016\/j.jtct.2026.04.006_bib0031","doi-asserted-by":"crossref","first-page":"2456","DOI":"10.3390\/ijms25052456","article-title":"Current strategies for increasing knock-in efficiency in CRISPR\/Cas9-based approaches","volume":"25","author":"Leal","year":"2024","journal-title":"Int J Mol Sci"},{"issue":"1","key":"10.1016\/j.jtct.2026.04.006_bib0032","doi-asserted-by":"crossref","DOI":"10.1016\/j.ymgme.2022.106968","article-title":"The landscape of CRISPR\/Cas9 for inborn errors of metabolism","volume":"138","author":"Leal","year":"2023","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jtct.2026.04.006_bib0033","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2023.101153","article-title":"Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice","volume":"31","author":"Leal","year":"2023","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.jtct.2026.04.006_bib0034","doi-asserted-by":"crossref","DOI":"10.3389\/fped.2022.877735","article-title":"The outcome of allogeneic hematopoietic stem cell transplantation from different donors in recipients with mucopolysaccharidosis","volume":"10","author":"Qu","year":"2022","journal-title":"Front Pediatr"},{"issue":"2","key":"10.1016\/j.jtct.2026.04.006_bib0035","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1016\/j.ymgme.2015.09.011","article-title":"Hematopoietic stem cell transplantation for Morquio A syndrome","volume":"117","author":"Yabe","year":"2016","journal-title":"Mol Genet Metab"},{"issue":"19","key":"10.1016\/j.jtct.2026.04.006_bib0036","doi-asserted-by":"crossref","first-page":"3981","DOI":"10.1182\/blood-2012-09-455238","article-title":"Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning","volume":"121","author":"Boelens","year":"2013","journal-title":"Blood"}],"container-title":["Transplantation and Cellular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S266663672600268X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S266663672600268X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,8,5]],"date-time":"2026-08-05T06:55:52Z","timestamp":1785912952000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S266663672600268X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8]]},"references-count":36,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2026,8]]}},"alternative-id":["S266663672600268X"],"URL":"https:\/\/doi.org\/10.1016\/j.jtct.2026.04.006","relation":{},"ISSN":["2666-6367"],"issn-type":[{"value":"2666-6367","type":"print"}],"subject":[],"published":{"date-parts":[[2026,8]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Long-Term Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Mucopolysaccharidosis IVA: A 10-Year Follow-Up from a Single-Center Experience in China","name":"articletitle","label":"Article Title"},{"value":"Transplantation and Cellular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jtct.2026.04.006","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights are reserved, including those for text and data mining, AI training, and similar technologies.","name":"copyright","label":"Copyright"}]}}