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Showing articles 0 to 4 of 4 Filter Applied: lysosomal storage disease (Click to remove) Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy NEJM 392:1609-1620, Fumagalli,F.,et al, 2025 AAV9-Mediated Gene Therapy for Infantile-Onset Pompes Disease NEJM 392:2438-2446, 2477, Ma,X.,et al, 2025 Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 Intrathecal 2-hydroxypropyl-�-cyclodextrin Decreases Neurological Disease Progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial Lancet 390:1758-1768, Ory, D.S.,et al, 2017 Showing articles 0 to 4 of 4