Niemann-Pick disease type C, severe early infantile neurologic onset
ORPHA: 2169751 Treatment Available
Overview
human disease
Available Treatments (1)
| Drug | Form | Status | Countries | Lead Time |
|---|---|---|---|---|
| miglustat Orphan | Oral capsule, 100mg | FDA Approved, EMA Approved | 9 | 14d |
Classification & Codes
Orphanet Code
ORPHA:216975Niemann-Pick disease type C, severe early infantile neurologic onset
| Orphanet | ORPHA:216975 |
| Treatments | 1 drug(s) |
| Status | published |
Treatment Summary
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO