Outcome, Enzyme Replacement Therapy, GALNS Protein, Human, Galsulfase, Idursulfase pp.247-252 発行日 2016年3月1日 Published Date 2016/3/1 DOI https://doi.org/10.18888/J00639.2016153583
[webview.isho.jp]
Treatment with enzyme replacement therapy has allowed the missing enzyme to be replaced with the aim to reduce the effects of the disease and prevent it progressing.
[cochrane.org]
Enzyme replacement therapy (ERT) has not been shown to be effective in MPS III.
[mpssociety.org]