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A phase I/II clinical trial of JR-441 for treatment of Sanfilippo syndrome type A (MPS IIIA)

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Bibliographic Details
Published in:Molecular genetics and metabolism 2024-02, Vol.141 (2), p.107973, Article 107973
Main Authors: Muschol, Nicole M., Lindschau, Mona, Quitmann, Christina, Hufschmidt, Johannes, O'Neill, Cara, van den Akker, Hanneke, Claros, Maria Angelica Cordoba, Kobayashi, Minako, Kawashima, Satoshi, Tanizawa, Kazunori, Schmidt, Mathias, Sato, Yuji, Kamitani, Ami
Format: Article
Language:English
Online Access:Get full text
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2023.107973