Journal of mucopolysaccharidosis and rare diseases

Association for Research of MPS and Rare Diseases (뮤코다당증연구학회)
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Development of New Strategies for Enzyme Replacement Therapy for Lysosomal Storage Disorders

  • Ko, Ah-Ra (Research Institute for Future Medicine, Samsung Biomedical Research Institute)
  • Received : 2016.05.30
  • Accepted : 2016.06.12
  • Published : 2016.06.30
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Abstract

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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References

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