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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 X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy JNNP 63:4-14, vanGeel,B.M.,et al, 1997 Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy NEJM 391:1256-1258, Laugwitz,L.,et al, 2024 Lentiviral Gene Therapy for Cerebral adrenoleukodystrophy NEJM 391:1302-1312,1358, Eichler,F.,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 Muscular Dystrophies Lancet 394:2025-2038, Mercuri, E.,et al, 2019 Tumor-free Survival of 7 Years After Gene Therapy for Recurrent Glioblastoma Neurol 61:270-271, Floeth,F.W,,et al, 2003 Gene Therapy for Neurologic Disease Arch Neurol 50:1252-1268, Suhr,S.T.&Gage,F.H., 1993 Showing articles 0 to 8 of 8