α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial-Reference-Cited by-同舟云学术

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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

Published:2024-03 Issue:3 Volume:32 Page:609-618
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

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^ORCID

Publisher

Elsevier BV

Reference19 articles.

1. Mucopolysaccharidosis type I: Clinical features, biochemistry, diagnosis, genetics, and treatment;Munoz-Rojas MV,2018

2. Mucopolysaccharidosis Type I (MPS I);Tylki-Szymanska,2022

3. Epidemiology of mucopolysaccharidoses;Khan;Mol. Genet. Metab.,2017

4. The natural history of MPS I: global perspectives from the MPS I Registry;Beck;Genet. Med.,2014

5. Mucopolysaccharidosis I: management and treatment guidelines;Muenzer;Pediatrics,2009

Cited by 1 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Transcytosis-Driven Treatment of Neurodegenerative Disorders by mRNA-Expressed Antibody–Transferrin Conjugates;Biomedicines;2024-04-12

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