Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy-Reference-Cited by-同舟云学术

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Activity of daily living in mucopolysaccharidosis IVA patients: Evaluation of therapeutic efficacy

Published:2021-10-08 Issue:11 Volume:9 Page:
ISSN:2324-9269
Container-title:Molecular Genetics & Genomic Medicine
language:en
Short-container-title:Mol Genet Genomic Med

Author:

Chen Hui¹²,Khan Shaukat²,Celik Betul¹²,Suzuki Yasuyuki³,Ago Yasuhiko⁴^ORCID,Tomatsu Shunji¹²⁴⁵^ORCID

Affiliation:

1. University of Delaware Newark DE USA

2. Nemours/Alfred I. duPont Hospital for Children Wilmington DE USA

3. Medical Education Development Center Graduate School of Medicine Gifu University Gifu Japan

4. Department of Pediatrics Graduate School of Medicine Gifu University Gifu Japan

5. Department of Pediatrics Thomas Jefferson University Philadelphia PA USA

Funder

Foundation for the National Institutes of Health

Publisher

Wiley

Subject

Genetics (clinical),Genetics,Molecular Biology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mgg3.1806

Reference73 articles.

1. UPLC-MS/MS detection of disaccharides derived from glycosaminoglycans as biomarkers of mucopolysaccharidoses

2. Tracheal narrowing in children and adults with mucopolysaccharidosis type IVA: evaluation with computed tomography angiography

3. Histoire naturelle de la mucopolysaccharidose IV de type A (maladie de Morquio A)

4. Respiratory and sleep disorders in mucopolysaccharidosis

5. Outcomes from 18 years of cervical spine surgery in MPS IVA: a single centre’s experience

Cited by 4 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Pain management challenges in a patient with mucopolysaccharidosis IVA;Clinical Case Reports;2024-07-28

2. Measuring health related quality of life (HRQoL) in Lysosomal Storage Disorders (LSDs): a rapid scoping review of available tools and domains;Orphanet Journal of Rare Diseases;2024-07-04

3. Neuropsychology assessment and outcomes in adult mucopolysaccharidosis – A systematic review as the first step to service development in a large tertiary Lysosomal Storage Disorders centre;Molecular Genetics and Metabolism;2023-02

4. Clinical characteristics and effects of enzyme replacement therapy with elosulfase alfa in Korean patients with mucopolysaccharidosis type IVA;Molecular Genetics and Metabolism Reports;2022-06

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