Browsing by Author "Eto, Yoshikatsu"

Now showing items 1-1 of 1

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 ...