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A Review Of The Clinical Outcomes In Idursulfase-Treated And Untreated Filipino Patients With Mucopolysaccharidosis Type II: Data From The Local Lysosomal Storage Disease Registry

2020-11-03

Abstract excerpt

<title>Abstract</title> <p><bold>Background:</bold> Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked multisystem disorder characterized by glycosaminoglycan (GAG) accumulation, caused by a deficiency of iduronate-2-sulfatase (I2S). Enzyme replacement therapy (ERT) with recombinant I2S, the standard of care, was started in the Philippines in 2017. This study reviewed the clinical outcomes in...

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Literature Corpus work: a7fd9d28-6af3-594b-b32f-f53a7c047dfa
DOI: 10.21203/rs.3.rs-98874/v1

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