A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II

Pabinafusp alfa (JR-141) is a novel enzyme drug that crosses the blood-brain barrier by transcytosis via transferrin receptors. In order to establish its efficacy and safety, a multicenter, single-arm, open-label phase 2/3 clinical trial was conducted in 28 Japanese patients with mucopolysaccharidos...

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Detalles Bibliográficos
Autores principales: Okuyama, Torayuki, Eto, Yoshikatsu, Sakai, Norio, Nakamura, Kimitoshi, Yamamoto, Tatsuyoshi, Yamaoka, Mariko, Ikeda, Toshiaki, So, Sairei, Tanizawa, Kazunori, Sonoda, Hiroyuki, Sato, Yuji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7854283/
https://www.ncbi.nlm.nih.gov/pubmed/33038326
http://dx.doi.org/10.1016/j.ymthe.2020.09.039

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7854283/
https://www.ncbi.nlm.nih.gov/pubmed/33038326
http://dx.doi.org/10.1016/j.ymthe.2020.09.039

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