Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey-Reference-Cited by-同舟云学术

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Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey

Published:2023-12 Issue: Volume:37 Page:101005
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Lau Heather,Harmatz Paul,Botha Jaco,Audi Jennifer,Link Bianca

Funder

Takeda Pharmaceuticals International

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference51 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Epidemiology of mucopolysaccharidoses;Khan;Mol. Genet. Metab.,2017

3. Mucopolysaccharidosis type II (hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy;Wraith;Eur. J. Pediatr.,2008

4. Prevalence and characterization of cardiac involvement in hunter syndrome;Kampmann;J. Pediatr.,2011

5. Orthopedic manifestations in patients with mucopolysaccharidosis type II (Hunter syndrome) enrolled in the Hunter Outcome Survey Orthop Rev (Pavia);Link,2010

Cited by 1 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. Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond;BioDrugs;2024-08-23

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