Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil

In Hunter syndrome (mucopolysaccharidosis II [MPS-II]), systemic accumulation of glycosaminoglycans (GAGs) due to a deficiency of iduronate-2-sulfatase (IDS), caused by mutations in the IDS gene, leads to multiple somatic manifestations and in patients with the severe (neuronopathic) phenotype, also...

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Detalles Bibliográficos
Autores principales: Giugliani, Roberto, Martins, Ana Maria, So, Sairei, Yamamoto, Tatsuyoshi, Yamaoka, Mariko, Ikeda, Toshiaki, Tanizawa, Kazunori, Sonoda, Hiroyuki, Schmidt, Mathias, 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/PMC8261166/
https://www.ncbi.nlm.nih.gov/pubmed/33781915
http://dx.doi.org/10.1016/j.ymthe.2021.03.019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8261166/
https://www.ncbi.nlm.nih.gov/pubmed/33781915
http://dx.doi.org/10.1016/j.ymthe.2021.03.019

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