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Showing articles 0 to 8 of 8 Filter Applied: lysosomal storage disease (Click to remove) Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy NEJM 392:1609-1620, Fumagalli,F.,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 Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome NEJM 385:1929-1940, Gentner, B.,et al, 2021 Pes Cavus and Neuropathy Neurol 93:e823-e826, Alderson,J.,& Ghosh,P.S., 2019 Adult-Onset Niemann-Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult Front Neurol doi.10.3398/jneur.2017.00108, Piroth,T.,et al, 2017 Small Fiber Neuropathy in Fabry Disease:A Review of Pathophysiology and Treatment JIEMS 4:1-5, Politeri,J.M.,et al, 2016 Psychosis in an Adolescent Girl: A Common Manifestation in Niemann-Pick Type C Disease Child Adolesc Pych Ment Health 8:20, Wouters,S.,et al, 2014 Enzyme Replacement Therapy With Agalsidase Alfa in Patients With Fabrys Disease: An Analysis of Registry Data Lancet 374:1986-1996, 1950, Mehta,A.,et al, 2009 Showing articles 0 to 8 of 8