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Systems-level investigation of mucopolysaccharidosis IIIA identifies deficient synaptic activity as a key driver of disease progression

Adrian Salavaty, Alon M Douek, Florian Kreuder, Sebastian-Alexander Stamatis, Joel R Steele, Iresha Hanchapola, Anup D Shah, Ralf B Schittenhelm, Mirana Ramialison, Peter D Currie, Jan Kaslin

Last updated on Dec 28, 2023

Systems-level investigation of mucopolysaccharidosis IIIA identifies deficient synaptic activity as a key driver of disease progression