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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

Document Type

Article

Author

Harmatz, Paul; Giugliani, Roberto; Martins, Ana Maria; 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

Source

In Molecular Therapy 6 March 2024 32(3):609-618

Subject

Language

ISSN

1525-0016

Online Access

Full Text (ScienceDirect 종량제) Web of Science JCR 저널정보 Scopus Find it@PNU

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