Enter your terms above and click the 'Search' button.

Showing articles 0 to 4 of 4 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 Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy NEJM 391:1256-1258, Laugwitz,L.,et al, 2024 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 "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 Showing articles 0 to 4 of 4