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Niemann-Pick disease type C, adult neurologic onset

MONDO:0016310

7 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Metabolic disease (233) Inherited lipid metabolism disorder (189) Hereditary disease (176) Immune system disorder (148) Inborn errors of metabolism (45) Lysosomal storage disease (35) Lymphatic system disorder (29) Histiocytosis (16) Human disease (14)

Trials to join now! 2 Not yet finished but already full! 4 Completed 1

New drug targets tough childhood cancers with genetic flaws

Disease control Completed

This phase 2 trial tested the drug samotolisib in 18 children and young adults with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that had spread, returned, or stopped responding to treatment. Only patients whose tumors had specific mutations in the TSC or…

Phase: PHASE2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control

Last updated Jun 27, 2026 12:23 UTC