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Showing articles 0 to 2 of 2 Filter Applied: storage disease of CNS (Click to remove) AAV9-Mediated Gene Therapy for Infantile-Onset Pompes Disease NEJM 392:2438-2446, 2477, Ma,X.,et al, 2025 Clinicopathologic Conference, Kufs Disease (Autosomal Dominant) Parry Type Neuronal Ceroid Lypofuscinosis NEJM 364:1062-1074, Case 8-2011, 2011 Showing articles 0 to 2 of 2