Nowadays, non-resolving inflammation is becoming a major trigger in various diseases as it
plays a significant role in the pathogenesis of atherosclerosis, asthma, cancer, obesity …

Nonsense suppression therapy encompasses approaches aimed at suppressing translation
termination at in-frame premature termination codons (PTCs, also known as nonsense …

Mucopolysaccharidosis type II (MPS II, Hunter syndrome) was first described by Dr. Charles
Hunter in 1917. Since then, about one hundred years have passed and Hunter syndrome …

Summary Mucopolysaccharidosis type III (MPS III, Sanfilippo syndrome) is an autosomal
recessive disorder, caused by a deficiency in one of the four enzymes involved in the …

Mucopolysaccharidoses (MPS) are a group of rare lysosomal storage diseases (LSD)
caused by genetic defects. These genetic defects lead to a lack or deficiency of enzymes …

Cystinosis is an autosomal recessive metabolic disease that belongs to the family of
lysosomal storage disorders. It is caused by a defect in the lysosomal cystine transporter …

Lysosomal storage diseases (LSDs) are a group of genetic disorders due to defects in any
aspect of lysosomal biology. During the past two decades, different approaches have been …

MPS disorders are associated with a wide spectrum of neurocognitive effects, from mild
problems with attention and executive functions to progressive and degenerative …

Mucopolysaccharidoses (MPSs) constitute a heterogeneous group of lysosomal storage
disorders characterized by the lysosomal accumulation of glycosaminoglycans (GAGs) …

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 …