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...

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Detalles Bibliográficos
Autores principales: Okuyama, Torayuki, Eto, Yoshikatsu, Sakai, Norio, Minami, Kohtaro, Yamamoto, Tatsuyoshi, Sonoda, Hiroyuki, Yamaoka, Mariko, Tachibana, Katsuhiko, Hirato, Tohru, Sato, Yuji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Original Article
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

Internet

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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