Niemann-Pick disease type C

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At a Glance

Live from database

Regulatory Watchboard

4 events
Nov 2022Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) — NA

TrialRECRUITING
Jun 2022A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

IntraBio Inc — PHASE3

TrialRECRUITING
Jul 2021Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

Cyclo Therapeutics, Inc. — PHASE3

TrialACTIVE NOT RECRUITING
Aug 2006Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) — NA

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

0

Total programs

No financial assistance programs currently listed for Niemann-Pick disease type C.
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Approved Treatments

1 FDA-approved

AQNEURSA

(LEVACETYLLEUCINE)Orphan drugstandard

IntraBio Inc

FDA label ↗

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Clinical Trial Landscape

4 active trials
2Phase 3
2N/A
4Total recruiting
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Recent News & Research

No recent news articles indexed yet for Niemann-Pick disease type C.
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Specialist Network

Top 6 by expertise

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