Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation-Reference-Cited by-同舟云学术

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

Published:2022-04-26 Issue:4 Volume:63 Page:371-378
ISSN:2192-8312
Container-title:JIMD Reports
language:en
Short-container-title:JIMD Reports

Author:

Maier Stefanie¹,Zivicnjak Miroslav²,Grigull Lorenz³,Hennermann Julia B.⁴,Aries Charlotte⁵,Maecker‐Kolhoff Britta¹,Sauer Martin¹,Das Anibh M.²,Beier Rita¹^ORCID

Affiliation:

1. Department of Paediatric Haematology and Oncology Hannover Medical School Hannover Germany

2. Department of Paediatric Kidney Liver and Metabolic Diseases at Hannover Medical School Hannover Germany

3. Rare Disease Centre, Bonn University Medical Centre Bonn Germany

4. Villa Metabolica, Department of Paediatric and Adolescent Medicine University Medical Centre Mainz Germany

5. Department of Paediatrics Hamburg‐Eppendorf University Medical Centre Hamburg Germany

Funder

Medizinischen Hochschule Hannover

Publisher

Wiley

Subject

Biochemistry, Genetics and Molecular Biology (miscellaneous),Endocrinology, Diabetes and Metabolism,Internal Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jmd2.12291

Reference35 articles.

1. Genotype‐phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I Registry

2. Variable clinical presentation in lysosomal storage disorders

3. Orthopaedic aspects of mucopolysaccharidoses

4. The natural history of neurocognition in MPS disorders: A review

5. Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

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1. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome;Science Translational Medicine;2024-05

2. MPSI Manifestations and Treatment Outcome: Skeletal Focus;International Journal of Molecular Sciences;2022-09-22