First in-human intracisternal dosing of RGX-111 (adeno-associated virus 9/human α-L-iduronidase) for a 20-month-old child with mucopolysaccharidosis type I (MPS I): 1 year follow-up

Autor: Wang, Raymond Y., Beydoun, Tammam, Movsesyan, Nina, Kan, Shih-Hsin, Taylor, Mery, Stockton, Winnie, Nestrasil, Igor, Pukenas, Bryan, Falabella, Paulo, Cho, Yoonjin, Névoret, Marie-Laure
Zdroj: In Molecular Genetics and Metabolism February 2021 132(2):S110-S110
Databáze: ScienceDirect
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