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Showing articles 0 to 9 of 9 Filter Applied: gene therapy (Click to remove) AAV9-Mediated Gene Therapy for Infantile-Onset Pompes Disease NEJM 392:2438-2446, 2477, Ma,X.,et al, 2025 Glycogen-Storage Disease Type II eMedicine, May 2, Ibrahim,J. &McGovern,M., 2006 Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy NEJM 392:1609-1620, Fumagalli,F.,et al, 2025 Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy NEJM 391:1256-1258, Laugwitz,L.,et al, 2024 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 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 X Linked Adrenoleukodystrophy:Clinical Presentation, Diagnosis, and Therapy JNNP 63:4-14, vanGeel,B.M.,et al, 1997 Showing articles 0 to 9 of 9