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

Abstract 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...

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Bibliographic Details
Main Authors: Stefanie Maier, Miroslav Zivicnjak, Lorenz Grigull, Julia B. Hennermann, Charlotte Aries, Britta Maecker‐Kolhoff, Martin Sauer, Anibh M. Das, Rita Beier
Format: Article
Language:English
Published: Wiley 2022-07-01
Series:JIMD Reports
Subjects:
Online Access:https://doi.org/10.1002/jmd2.12291