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. Author manuscript; available in PMC: 2020 Jul 2.
Published in final edited form as: Gene Ther. 2020 Jan 2;27(5):226–236. doi: 10.1038/s41434-019-0120-5

Table 1:

Previous preclinical studies show that high doses of ERT can treat neurological complications.

Mouse model Dose (mg/kg) Clinical dose (mg/kg) Brain enzyme activity (% Normal) Brain storage reduced (%Affected) References
α-mannosidosis 36.6 1 15% 50% [27]
Metachromatic leukodystrophy 20 N/A N/A 30% [26]
Aspartylglycosaminuria 10 N/A 10% 20% [28]
Krabbe disease 6 N/A 7% 18% [25]
MPS II 10 0.5 5% N/A [22]
MPS IIIA 20 N/A 22% 0 [23]
MPS VII 20 2 2.50% N/A [24]
MPS I 20 0.58 97% 63% [21]