Browsing Biological Sciences by Author "Takasao, Naoko"
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α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: a phase 1/2 trial
Harmatz, Paul; Giugliani, Roberto; Martins, Ana Maria (Medicina); Hamazaki, Takashi; Kubo, Toru; Kira, Ryutaro; Minami, Kohtaro; Ikeda, Toshiaki; Moriuchi, Hiroaki; Kawashima, Satoshi; Takasao, Naoko; So, Sairei; Sonoda, Hiroyuki; Hirato, Tohru; Tanizawa, Kazunori; Schmidt, Mathias; Sato, Yuji (2024) [Journal article]Mucopolysaccharidosis type I (MPS I) causes systemic accumulation of glycosaminoglycans due to a genetic deficiency of α-L-iduronidase (IDUA), which results in progressive systemic symptoms affecting multiple organs, ...