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A Phase 2/3 Trial of Pabinafusp Alfa,IDS Fused with Anti-Human Transferrin Receptor Antibody,Targeting Neurodegeneration in MPS-II |
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| Authors: | Torayuki Okuyama Yoshikatsu Eto Norio Sakai Kimitoshi Nakamura Tatsuyoshi Yamamoto Mariko Yamaoka Toshiaki Ikeda Sairei So Kazunori Tanizawa Hiroyuki Sonoda Yuji Sato |
| Affiliation: | 1. National Center for Child Health and Development, Tokyo 157-8535, Japan;2. Advanced Clinical Research Center and Asian Lysosome Storage Disorder Center, Institute of Neurological Disorders, Kawasaki 215-0026, Japan;3. Osaka University, Course of Health Science, Osaka 565-0871 Japan;4. Kumamoto University Graduate School of Medical Science, Kumamoto 860-0811, Japan;5. JCR Pharmaceuticals, Ashiya 659-0021, Japan |
| Abstract: |  |
| Keywords: | mucopolysaccharidosis II Hunter syndrome pabinafusp alfa blood brain barrier iduronate-2-sulfatase central nervous system neurocognitive development neurocognitive impairment neurodegeneration anti-human transferrin receptor antibody heparan sulfate enzyme-replacement therapy |
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