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Autor:
Estera Rintz, Magdalena Podlacha, Zuzanna Cyske, Karolina Pierzynowska, Grzegorz Węgrzyn, Lidia Gaffke
Publikováno v:
Neurotherapeutics. 20:254-271
Sanfilippo disease, caused by mutations in the genes encoding heparan sulfate (HS) (a glycosaminoglycan; GAG) degradation enzymes, is a mucopolysaccharidosis (MPS), which is also known as MPS type III, and is characterized by subtypes A, B, C, and D,