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...
Main Authors: | , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Wiley
2022-07-01
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Series: | JIMD Reports |
Subjects: | |
Online Access: | https://doi.org/10.1002/jmd2.12291 |