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Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial
Hunter syndrome (mucopolysaccharidosis II [MPS II]), a deficiency of iduronate-2-sulfatase (IDS), causes an accumulation of glycosaminoglycans, giving rise to multiple systemic and CNS symptoms. The currently available therapies, idursulfase and idursulfase beta, are ineffective against the CNS symp...
Autores principales: | Okuyama, Torayuki, Eto, Yoshikatsu, Sakai, Norio, Minami, Kohtaro, Yamamoto, Tatsuyoshi, Sonoda, Hiroyuki, Yamaoka, Mariko, Tachibana, Katsuhiko, Hirato, Tohru, Sato, Yuji |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6391590/ https://www.ncbi.nlm.nih.gov/pubmed/30595526 http://dx.doi.org/10.1016/j.ymthe.2018.12.005 |
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