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Showing articles 0 to 3 of 3 Filter Applied: gene therapy (Click to remove) Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy NEJM 391:1256-1258, Laugwitz,L.,et al, 2024 X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy JNNP 63:4-14, vanGeel,B.M.,et al, 1997 Current and Emerging Issues in Wilsons Disease NEJM 389:922-938, Roberts,E.A. & Schilsky, M.L., 2023 Showing articles 0 to 3 of 3