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 Teduced (% Affected) | References |
|---|---|---|---|---|---|
| α-Mannosidosis | 18.3 | 1 | N/A | 74 | 14 |
| α-Mannosidosis | 36.6 | 1 | 15 | 50 | 15 |
| Metachromatic leukodystrophy | 20 | N/A | N/A | 30 | 16 |
| Metachromatic leukodystrophy | 50 | N/A | N/A | 34 | 17 |
| Aspartylglycosaminuria | 10 | N/A | 10 | 20 | 18 |
| Krabbe disease | 6 | N/A | 7 | 18 | 19 |
| MPS II | 10 | 0.5 | 5 | N/A | 20 |
| MPS IIIA | 20 | N/A | 22 | 0 | 21 |
| MPS VII | 20 | 2 | 2.50 | N/A | 22 |
| MPS I | 20 | 0.58 | 97 | 63 | 23 |
N/A, not available.