Associated Papers
Enzyme Replacement Therapy in Mucopolysaccharidosis Type VII: A Three-Year Clinical Outcome Study of the First Taiwanese Case.
Lee CL et al.
Diagnostics (basel, Switzerland), 2025
Clinical manifestations and genetic mutation analysis of patients with mucopolysaccharidosis type VII in China.
Su X et al.
European Journal of Medical Genetics, 2024
The first mucopolysaccharidosis type VII in a Taiwanese girl: A case report and review of the literature.
Lee CL et al.
Journal of the Formosan Medical Association = Taiwan Yi Zhi, 2022
First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient.
Fox JE et al.
Molecular Genetics and Metabolism, 2015
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