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- 학술저널
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- 뮤코다당증연구학회 Journal of mucopolysaccharidosis and rare disease Journal of mucopolysaccharidosis and rare disease 제2권 제1호
- 발행연도
- 2016.1
- 수록면
- 17 - 18 (2page)
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Enzyme replacement therapy (ERT) is a well-established means of treating lysosomal storage disease (LSD). However, classical IV infusion based ERT method produces less than ideal results, especially, CNS defects and quality of life in patients. To improve these main problems of parental IV formulation for LSDs, we investigate modified ERT method and evaluated the efficacy in animal model.
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