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2026
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""Gene Therapy Agents""[mesh_terms]

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Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.

John J Mitchell, Jose E Abdenur, Foekje de Boer, Monica Boyer, Margo Sheck Breilyn, María-Luz Couce, Diva D De Leon, Terry G Derks, Areeg El-Gharbawy, Andrea B Haijer-Schreuder, Karen Loechner, Nicola Longo, Allan M Lund, Miguel Angel Martinez Olmos, Shawn E McCandless, Bibiana Mello de Oliveira, Malaya Mount, Nicole Muschol, Kristina Pytlak, Kadakkal Radhakrishnan, Rebecca Riba-Wolman, David F Rodriguez-Buritica, Alessandro La Rosa, Alessandro Rossi, Heather Saavedra, René Santer, Brian Shayota, G Peter A Smit, Carolina F Moura De Souza, Melanie M van der Klauw, David A Weinstein, Joseph I Wolfsdorf, Anne Blake, Andrew A Grimm, Deepali Mitragotri, Syeda Rahman, Diane M Turner-Bowker, Richard Collis

Journal of inherited metabolic disease 2026;49(5):e70241

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