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Showing articles 0 to 6 of 6 Filter Applied: efficacy (Click to remove) Long-Term Benefit of Enzyme Replacement Therapy in Pompe Disease Neurol 89:2365-2373, Kuperus, E.,et al, 2017 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 Evidence-based Guideline: Intravenous Immunoglobulin in the Treatment of Neuromascular Disorders Neurol 78:1009-1015, Patwa,H.S.,et al, 2012 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 SLC01B1 Variants and Statin-Induced Myopathy -- A Genomewide Study NEJM 359:789-799, The SEARCH Collaborative Group, 2008 Showing articles 0 to 6 of 6