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Showing articles 0 to 8 of 8 Filter Applied: gene therapy (Click to remove) Muscular Dystrophies Lancet 394:2025-2038, Mercuri, E.,et al, 2019 Parkinsons Disease NEJM 391:442-452, Tanner,C.M. & Ostrem,J.L., 2024 Lentiviral Gene Therapy for Cerebral adrenoleukodystrophy NEJM 391:1302-1312,1358, Eichler,F.,et al, 2024 Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 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 Showing articles 0 to 8 of 8