Mucopolysaccharidosis I therapeutic (bluebird bio)

Mucopolysaccharidosis type I gene Therapy(Cellectis)

SGSH gene therapy (AAV9, mucopolysaccharidosis IIIA)

IDUA gene therapy (AAV9, mucopolysaccharidosis I)

GALNS gene therapy (AAV8, Mucopolysaccharidosis IVA)

AAV-TT coHGSNAT gene therapy (intracerebral, mucopolysaccharidosis IIIC)

triple capsid mutant based NAGLU gene therapy (AAV8, Mucopolysaccharidosis type IIIB)

Vestronidase alfa(Mepsevii)

Rx

Elosulfase alfa(Vimizim)

Rx

Laronidase(Aldurazyme)

Rx