Table 1.
LSDs | Affected Enzyme | Available ERT | Development Status | Reference |
---|---|---|---|---|
GD | Glucocerebrosidase | Imglucerase | Approved | [18] |
Velaglucerase | Approved | |||
Taliglucerase alpha | Approved | |||
FD | α-Galactosidase A | Agalsidase alpa | Approved | [19] |
Agalsidase beta | Approved | NCT03018730 | ||
PRX-102 | Phase III | NCT02795676 | ||
NCT03180840 | ||||
PD | α-Glycosidase | Alglucosidase alpha | Approved | [20] |
Avalglucosidase alfa | Approved | [21] | ||
VAL-1221 | Phase I-II | NCT02898753 | ||
ATB200 | Phase III | NCT03729362 | ||
MPS I | α-L-iduronidase | Laronidase | Approved | [22] |
MPS II | Iduronate-2-sulfatase | Idursulfasa | Approved | [23] |
AGT-182 | Phase I | NCT02262338 | ||
JR-141 | Approved | [24] | ||
MPS III
A B |
Heparan N-sulphatase | rhHNS | Phase I-II | NCT01299727 |
N-acetyl-glucosaminidase | BMN250 | Phase I-II | NCT02754076 | |
MPS VI | N-acetylgalactosamine-4-sulfatase | Galsufase | Approved | [24] |
MLD | Arilsulphatase A | HGT1110 | Phase I-II | NCT01887938 |
TAK611 | Phase II | NCT015128 [25] | ||
NPA and NPB |
Acid
Sphingomyelinase |
rh-ASM | Phase I | NCT00410566 |