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386 results for “NPC1”

Trials

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Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

66 participants · First posted Jul 12, 2021

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

56 participants · First posted Aug 28, 2015

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

13 participants · First posted Oct 20, 2016

Drugs

View all 55 →

AAV9-CaMKII-NPC1

AAV9-EF1S-NPC1

APC1

News

View all 28 →

Rafael Holdings Reports 78% Success Rate for NPC1 Treatment in Phase 3 Pediatric Sub-Study

Trappsol Cyclo Shows Promise in Stabilizing or Improving Niemann-Pick Disease Type C1 in Young Patients

Rafael Holdings Completes $25 Million Rights Offering to Fund Trappsol Cyclo Phase 3 Trial for Rare Disease

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