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Showing articles 0 to 7 of 7 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 Spinal Muscular Atrophy A Timely Review Arch Neurol 68:979-984, Kolb, S.J.,et al, 2011 Diagnosis and New Treatments in Muscular Dystrophies JNNP 80:706-714, Manzur,A.Y. &Muntoni,F., 2009 Glycogen-Storage Disease Type II eMedicine, May 2, Ibrahim,J. &McGovern,M., 2006 Muscular Dystrophies Lancet 394:2025-2038, Mercuri, E.,et al, 2019 Diagnosis and Therapy in Neuromuscular Disorders: Diagnosis and New Treatments in Mitochondrial Diseases JNNP 80:943-953, Rahman,S. &Hanna,M.G., 2009 X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy JNNP 63:4-14, vanGeel,B.M.,et al, 1997 Showing articles 0 to 7 of 7