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Clinical characteristics of NPC1 patients and healthy control subjects included in this cohort.
| NPC1 untreated | NPC1 MGS treated | Heterozygous parents | Healthy controls | |
|---|---|---|---|---|
| Number (male/female/unknown) | 75 (42/33) | 89 (54/35) | 31 (15/13/3) | 30 (23/6/2) |
| Average age in years (range) | 19 (0.3–54) | 11 (1–28) | N/A | 15 (0.4–50) |
| ASIS range | 0.13–5.97 | 0.07–9.16 | N/A | N/A |
