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Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation

Mucopolysaccharidosis type I (MPS I) is an autosomal‐recessive metabolic disorder caused by an enzyme deficiency of lysosomal alpha‐l‐iduronidase (IDUA). Haematopoietic stem cell transplantation (HSCT) is the therapeutic option of choice in MPS I patients younger than 2.5 years, which has a positive...

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Detalles Bibliográficos
Autores principales:	Maier, Stefanie, Zivicnjak, Miroslav, Grigull, Lorenz, Hennermann, Julia B., Aries, Charlotte, Maecker‐Kolhoff, Britta, Sauer, Martin, Das, Anibh M., Beier, Rita
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2022
Materias:	Research Reports
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9259397/ https://www.ncbi.nlm.nih.gov/pubmed/35822096 http://dx.doi.org/10.1002/jmd2.12291

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