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Showing articles 0 to 8 of 8 Filter Applied: gene therapy (Click to remove) Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy NEJM 391:1256-1258, Laugwitz,L.,et al, 2024 Lentiviral Haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access Lancet 399:372-383, Fumagalli, F.,et al, 2022 Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy JNNP 63:4-14, vanGeel,B.M.,et al, 1997 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 Glycogen-Storage Disease Type II eMedicine, May 2, Ibrahim,J. &McGovern,M., 2006 Showing articles 0 to 8 of 8