Article

The usage of enzyme replacement treatments, economic burden, and quality of life of patients with four lysosomal storage diseases in Shanghai, China

2021-01-07

Abstract excerpt

<title>Abstract</title> <p>Background Lysosomal storage diseases (LSDs) are a group of rare diseases that caused progressive physical dysfunction and organ failure, which significantly affected patients’ quality of life. Enzyme replacement treatments (ERTs) are now acknowledged as the advanced therapies for LSDs while cost millions per patient per year. Previous studies seldom reported the usage of ERTs and dise...

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Literature Corpus work: f1b178a7-a88d-5c4f-89c7-bcb277154ba0
DOI: 10.21203/rs.3.rs-139895/v1

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