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Showing articles 0 to 7 of 7 Filter Applied: lysosomal storage disease (Click to remove) A New Metabolite Contributing to N-Acetyl Signal in 1H MRS of the Brain in Salla Disease Neurol 52:1668-1672, Varho,T.,et al, 1999 Disease-Modifying, Neuroprotective Effect of N-Acetyl-L-Leucine in Adult and Pediatric Patients with Neimann-Pick Disease Type C Neurol 105:e213589, Patterson,M.C., et al, 2025 AAV9-Mediated Gene Therapy for Infantile-Onset Pompes Disease NEJM 392:2438-2446, 2477, Ma,X.,et al, 2025 A 65-Year-Old Woman with Isolated Macroglossia as the Initial Presentation of a Rare Disease Neurol 103:e210070, Lara,C.,et al, 2024 "Im fine; Im just waiting for my disease" The New and Growing Class of Presymptomatic Patients Neurol 77:522-523, Kwon, J.M.et al, 2011 Glycogen-Storage Disease Type II eMedicine, May 2, Ibrahim,J. &McGovern,M., 2006 Clinicopath Conf, Neuronal Ceroid Lipofuscinosis, Late-Onset Infantile Subtype NEJM 347:672-680, Case 27-2002, 2002 Showing articles 0 to 7 of 7