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Showing articles 0 to 3 of 3 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 Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 Enzyme Replacement Therapy With Agalsidase Alfa in Patients With Fabrys Disease: An Analysis of Registry Data Lancet 374:1986-1996, 1950, Mehta,A.,et al, 2009 Showing articles 0 to 3 of 3