Lysosomal storage disorders (LSDs)—designated as' orphan'diseases—are inborn errors of
metabolism caused by defects in genes that encode proteins involved in various aspects of …

Lysosomal storage diseases (LSDs) are a group of rare, life-threatening genetic disorders,
usually caused by a dysfunction in one of the many enzymes responsible for intralysosomal …

Mucopolysaccharide diseases (MPS) are caused by deficiency of glycosaminoglycan (GAG)
degrading enzymes, leading to GAG accumulation. Neurodegenerative MPS diseases …

During the past decades, several therapeutic approaches have been developed and made
rapidly available for many patients afflicted with lysosomal storage disorders (LSDs), inborn …

Alzheimer's disease is one of severe neurological diseases for which no effective treatment
is currently available. The use of genistein (5, 7-dihydroxy-3-(4-hydroxyphenyl)-4H-1 …

Sanfilippo syndrome, or mucopolysaccharidosis (MPS) type III, refers to one of five
autosomal recessive, neurodegenerative lysosomal storage disorders (MPS IIIA to MPS IIIE) …

Genistein (5, 7-dihydroxy-3-(4-hydroxyphenyl)-4H-1-benzopyran-4-one) has been
previously proposed as a potential drug for use in substrate reduction therapy for …

Background Neurodegenerative metabolic disorders such as mucopolysaccharidosis IIIB
(MPSIIIB or Sanfilippo disease) accumulate undegraded substrates in the brain and are …

The accumulation of undegraded molecular material leads to progressive
neurodegeneration in a number of lysosomal storage disorders (LSDs) that are caused by …

Autophagy is a process of degradation of macromolecules in the cytoplasm, particularly
proteins of a long half-life, as well as whole organelles, in eukaryotic cells. Lysosomes play …