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Showing articles 0 to 8 of 8 Filter Applied: gene therapy (Click to remove) 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 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 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 Gene Therapy for Cerebral Vascular Disease Stroke 27:1688-1693, Heistad,D.D.&Faraci,F.M., 1996 Gene Therapy for Neurologic Disease Arch Neurol 50:1252-1268, Suhr,S.T.&Gage,F.H., 1993 Showing articles 0 to 8 of 8