Safety and Efficacy of DTX401 , an AAV8 ‐Mediated Liver‐Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a ( GSDIa )

Fuente: Wiley Open Access
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Bibliographic Details
Main Authors: David A. Weinstein, Terry G. Derks, David F. Rodriguez‐Buritica, Ayesha Ahmad, María‐Luz Couce, John J. Mitchell, Rebecca Riba‐Wolman, Malaya Mount, Julieta Bonvin Sallago, Katalin M. Ross, Melanie M. van der Klauw, Foekje de Boer, Caroline van der Schaaf, Heather Saavedra, Miguel Martínez‐Olmos, Elvis Atanga, Asad Hosseini, Deepali Mitragotri, Eric Crombez
Format: Artículo Open Access
Published: Wiley 2025
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