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Showing articles 0 to 8 of 8 Filter Applied: stem cell transplantation (Click to remove) Clinicopathologic Features, Pathogenesis, and Treatment of Monoclonal Gammopathy-Associated Myopathies Neurol 105:213101, Soontrapa,P.,et al, 2025 Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy NEJM 391:1256-1258, Laugwitz,L.,et al, 2024 A 23-Year-Old Woman Presenting with Cognitive Impairment and Gait Disturbance Neurol 99:997-1003, Chaity,D.K.,et al, 2022 Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 A 64-year-old Man with Progressive Paraspinal Muscle Weakness Neurol 86:e4-e9, Schneider, R.,et al, 2016 Stem Cell Transplantation in a Patient With Late-Onset Nemaline Myopathy and Gammopathy Neurol 71:531-532, Benveniste,O.,et al., 2008 Sporadic Late-Onset Nemaline Myopathy Effectively Treated by Melphalan and Stem Cell Transplant Neurol 71:532-534,472, Voermans,N.C.,et al., 2008 Insights Into the Diagnosis and Treatment of Lysosomal Storage Diseases Arch Neurol 60:322-328, Wegner,D.A.,et al, 2003 Showing articles 0 to 8 of 8