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Intrathecal Gene Therapy for Giant Axonal Neuropathy
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The New England Journal of Medicine
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
Intrathecal Gene Therapy for Giant Axonal Neuropathy
N. Engl. J. Med 2024 Mar 21;390(12)1092-1104, DX Bharucha-Goebel, JJ Todd, D Saade, G Norato, M Jain, T Lehky, RM Bailey, JA Chichester, R Calcedo, D Armao, AR Foley, P Mohassel, E Tesfaye, BP Carlin, B Seremula, M Waite, WM Zein, LA Huryn, TO Crawford, CJ Sumner, A Hoke, JD Heiss, L Charnas, JE Hooper, TW Bouldin, EM Kang, D Rybin, SJ Gray, CG BönnemannFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.