α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial
| Autor: | 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 |
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| Zdroj: | In Molecular Therapy 6 March 2024 32(3):609-618 |
| Databáze: | ScienceDirect |
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