alpha-N-acetylglucosaminidase

Recombinant alpha-N-acetylglucosaminidase (NAGLU) is an enzyme replacement therapy (ERT) being developed by TEGA Therapeutics for the treatment of Mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B. MPS IIIB is a rare, autosomal recessive lysosomal storage disorder caused by a deficiency in the NAGLU enzyme, which is essential for the degradation of the glycosaminoglycan heparan sulfate. The resulting accumulation of heparan sulfate leads to progressive and severe neurodegeneration, intellectual disability, and various systemic symptoms. This therapeutic approach involves the administration of a recombinant version of the missing enzyme to restore lysosomal function and facilitate the breakdown of accumulated substrates, with the goal of slowing or halting disease progression.

Other names

NAGLUrecombinant alpha-N-acetylglucosaminidaserecombinant NAGLU