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Showing articles 0 to 4 of 4 Filter Applied: gene therapy (Click to remove) Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy NEJM 392:1609-1620, Fumagalli,F.,et al, 2025 Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy NEJM 391:1256-1258, Laugwitz,L.,et al, 2024 Current and Emerging Issues in Wilsons Disease NEJM 389:922-938, Roberts,E.A. & Schilsky, M.L., 2023 Spinal Muscular Atrophy A Timely Review Arch Neurol 68:979-984, Kolb, S.J.,et al, 2011 Showing articles 0 to 4 of 4