Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Lysosomal storage diseases (LSDs) are a class of metabolic disorders caused by mutations
in proteins critical for lysosomal function. Such proteins include lysosomal enzymes …

The greatest challenge in developing therapies for mucopolysaccharidosis (MPS) IIIB is to
achieve efficient central nervous system (CNS) delivery across the blood–brain barrier …

Impaired degradation of glycosaminoglycans (GAGs) with consequent intralysosomal
accumulation of undegraded products causes a group of lysosomal storage disorders …

Abstract Mucopolysaccharidosis type III (MPS III) or Sanfilippo disease is an orphan
inherited lysosomal storage disease and one of the most common MPS subtypes. The …

Rare monogenic disorders such as lysosomal diseases have been at the forefront in the
development of novel treatments where therapeutic options are either limited or unavailable …

Mucopolysaccharidosis IIIB is a paediatric lysosomal storage disease caused by deficiency
of the enzyme α-N-acetylglucosaminidase (NAGLU), involved in the degradation of the …

Mucopolysaccharidosis IIIA (MPS IIIA or Sanfilippo disease) is a neurodegenerative disorder
caused by a deficiency in the lysosomal enzyme sulfamidase (SGSH), catabolizing heparan …

The mucopolysaccharidosis (MPS) disorders are a group of lysosomal storage diseases
caused by lysosomal enzyme deficits that lead to glycosaminoglycan accumulation, affecting …

No treatment is currently available for mucopolysaccharidosis (MPS) IIIB, a neuropathic
lysosomal storage disease caused by autosomal recessive defect in α-N …