Table 1:
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] |