Effects of Trehalose Administration in Patients with Mucopolysaccharidosis Type III.
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DOI: 10.2174/0929867330666230406102555
Abstract: BACKGROUND AND AIM 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 central… read more here.
Keywords: administration patients; mucopolysaccharidosis type; type iii; iii ... See more keywords