Browsing College of Medicine by Subject "Sanfilippo syndrome"
Now showing items 1-1 of 1
-
Effects of Trehalose Administration in Patients with Mucopolysaccharidosis Type III.
( Europe PMC , 2023 , Article)Mucopolysaccharidosis type III (MPS III) is a rare autosomal recessive lysosomal storage disease (LSD) caused by a deficiency of lysosomal enzymes required for the catabolism of glycosaminoglycans (GAGs), mainly in the ...