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Showing articles 0 to 2 of 2 Filter Applied: gene therapy (Click to remove) Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy NEJM 377:1713-1722,1786, Mendell, J.R.,et al, 2017 X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy JNNP 63:4-14, vanGeel,B.M.,et al, 1997 Showing articles 0 to 2 of 2