Browsing by Subject "Mucopolysaccharidosis type IIIA"
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An observational, prospective, multicenter, natural history study of patients with mucopolysaccharidosis type IIIA
(2022) [Journal article]Mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome) is a rare genetic lysosomal storage disease characterized by early and progressive neurodegeneration resulting in a rapid decline in cognitive ...