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. 2021 Nov 26;11(12):1775. doi: 10.3390/biom11121775

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