Review Article

Glycosaminoglycan Storage Disorders: A Review

Table 2

Available therapeutic approaches for mucopolysaccharidoses.

Pathology SubtypeAvailable therapeutic approaches

MPS IHurler (H)HSCT (recommended before 2 years of age)
Hurler -Scheie (H/S)ERT with Aldurazyme (laronidase; recombinant human α-L-iduronidase)
Scheie (S)ERT with Aldurazyme (laronidase; recombinant human α-L-iduronidase)
MPS IIHunterERT with Elaprase (idursulfase; recombinant human iduronate sulfatase)
MPS IIISanfilippo A Not available*
Sanfilippo B *Not available*
Sanfilippo C *Not available*
Sanfilippo D *Not available*
MPS IVMorquio AERT: ongoing clinical trial (with recombinant human GALNS)
Morquio B *Not available*
MPS VI (Maroteaux-Lamy)ERT with Naglazyme (galsulfase; recombinant human arylsulfatase B)
MPS VII (Sly) *Not available*
MPS IX *Not available*