Niemann-Pick disease type C, severe perinatal form

ORPHA: 216972

Overview

human disease

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Classification & Codes

Orphanet Code

ORPHA:216972
Niemann-Pick disease type C, severe perinatal form
OrphanetORPHA:216972
Treatments0 drug(s)
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO