Niemann-Pick disease type C

MONDO:0018982

NPC is a complex lipid storage disease mainly characterized by the accumulation of unesterified cholesterol in the late endosomal/lysosomal compartment.

Also known as: NPC, Niemann Pick Disease Type C

19 clinical trials for this condition and its sub-types, 12 tagged with Niemann-Pick disease type C itself.

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Where it sits in the disease tree

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Part of

↑ Autosomal recessive disease (999) ↑ Niemann-Pick disease (22)

Sub-types of Niemann-Pick disease type C

Niemann-Pick disease, type C1 2 trials
Niemann-Pick disease, type C2 1 trial
Niemann-Pick disease type C, adult neurologic onset 0 trials
Niemann-Pick disease type C, juvenile neurologic onset 0 trials
Niemann-Pick disease type C, late infantile neurologic onset 0 trials
Niemann-Pick disease type C, severe early infantile neurologic onset 0 trials
Niemann-Pick disease type C, severe perinatal form 0 trials

Including sub-types (19) Tagged with Niemann-Pick disease type C (12)

Trials to join now! 7 Not yet finished but already full! 2 Completed 2 Terminated 1

Niemann-Pick disease type C

Where it sits in the disease tree

New pill shows promise for rare brain disorders in early trial

Promising drug tested for rare liver disease in newborns