Sanfilippo disease is one of mucopolysaccharidoses (MPS), a group of lysosomal storage diseases characterized by build up of partially degraded glycosaminoglycans (GAGs). this element, and clinical tests are being carried out with enzyme alternative therapy, gene therapy, and substrate reduction therapy. These recent achievements are summarized and discussed with this review. (coding for heparan N-sulfatase),… Continue reading Sanfilippo disease is one of mucopolysaccharidoses (MPS), a group of lysosomal