Table 1.
Available ERT based medicaments for LSDs. In the reference column there are indicated either reference articles or clinical trial numbers referring to www.clinicaltrials.gov (accessed on 15 November 2021) web page.
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 |