Lysosomal Storage Disorders

Storage disorders are manifestations of lysosomal dysfunction usually as a consequence of defective single genes encoding for a single enzyme required for the metabolism of lipids, glycoproteins, or so-called mucopolysaccharides. Estimates indicate that approximately one in every 25,000 babies born in the United States will have some form of the mucopolysaccharidoses (see factsheet), a group of congenital disorders … Continue reading Lysosomal Storage Disorders