Browsing by Author "Ikeda, Toshiaki"

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Enzyme replacement therapy with pabinafusp alfa for neuronopathic mucopolysaccharidosis II : an integrated analysis of preclinical and clinical data

Giugliani, Roberto; Martins, Ana Maria (Medicina); Okuyama, Torayuki; Eto, Yoshikatsu; Sakai, Norio; Nakamura, Kimitoshi; Morimoto, Hideto; Minami, Kohtato; Yamamoto, Tatsuyoshi; Yamaoka, Mariko; Ikeda, Toshiaki; So, Sairei; Tanizawa, Kazunori; Sonoda, Hiroyuki; Schmidt, Mathias; Sato, Yuji (2021) [Journal article]

Enzyme replacement therapy (ERT) improves somatic manifestations in mucopolysaccharidoses (MPS). However, because intravenously administered enzymes cannot cross the blood–brain barrier (BBB), ERT is ineffective against ...
Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II : a phase 2 trial in Brazil

Giugliani, Roberto; Martins, Ana Maria (Medicina); So, Sairei; Yamamoto, Tatsuyoshi; Yamaoka, Mariko; Ikeda, Toshiaki; Tanizawa, Kazunori; Sonoda, Hiroyuki; Schmidt, Mathias; Sato, Yuji (2021) [Journal article]

In Hunter syndrome (mucopolysaccharidosis II [MPS-II]),systemic accumulation of glycosaminoglycans (GAGs) dueto a deficiency of iduronate-2-sulfatase (IDS), caused by mu-tations in theIDSgene, leads to multiple somatic ...