Niemann-Pick Disease, Type C1

DBCOND0060907Brain DiseasesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Niemann-Pick Disease, Type C1 is the indication for 2 drugs in DrugBank. It is covered by 2 curated, structured indications.

Also known as
Niemann-Pick Disease, Type C · Niemann-Pick Type C Disease · Niemann-Pick Disease Type C · Niemann-Pick Disease Type C1
Classified under
Indicated drugs

Resolves to

Vocabularies
MeSHM0335789ICD-10E75.242SNOMED CT18927009UMLSC0220756

What you can answer from here — as of October 25, 2026

2Indicated drugsWith each drug's targets and mechanism
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2Structured indicationsPopulation, line of therapy, route and combination as fields, not prose
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28Clinical trialsPhase, status and sponsor resolved per trial
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2Drugs reporting it as an adverse effectWith incidence where the label states it
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Which approved drugs for Niemann-Pick Disease, Type C1 share a target, and which companies have late-stage candidates against it?

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