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Showing articles 0 to 9 of 9 Filter Applied: gene therapy (Click to remove) Current and Emerging Issues in Wilsons Disease NEJM 389:922-938, Roberts,E.A. & Schilsky, M.L., 2023 Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 Muscular Dystrophies Lancet 394:2025-2038, Mercuri, E.,et al, 2019 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 Diagnosis and Therapy in Neuromuscular Disorders: Diagnosis and New Treatments in Mitochondrial Diseases JNNP 80:943-953, Rahman,S. &Hanna,M.G., 2009 Glycogen-Storage Disease Type II eMedicine, May 2, Ibrahim,J. &McGovern,M., 2006 X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy JNNP 63:4-14, vanGeel,B.M.,et al, 1997 Myoblast Transfer in Duchenne Muscular Dystrophy Ann Neurol 34:8-18, Karpati,G.,et al, 1993 Showing articles 0 to 9 of 9