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Orphazyme Reports Encouraging Arimoclomol Clinical Trial Top-Line Data in Niemann-Pick Disease Type C (NPC)
Copenhagen, September 28, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced encouraging top-line results for its clinical Phase II/III trial with orally... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - September 28, 2018 Category: Pharmaceuticals Source Type: clinical trials

Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
Condition:   Niemann-Pick Disease, Type C Intervention:   Drug: VTS-270 Sponsor:   Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - September 27, 2018 Category: Research Source Type: clinical trials

CTD Holdings Discusses its Phase I Clinical Trial for Intravenous Use of Trappsol ® Cyclo™ in Niemann-Pick Disease Type C
ALACHUA, Fla., Aug. 30, 2018 (GLOBE NEWSWIRE) -- CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, responds today to community... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - August 30, 2018 Category: Pharmaceuticals Source Type: clinical trials

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Condition:   Niemann-Pick Disease, Type C Intervention:   Drug: 2-Hydroxypropyl-Beta-Cyclodextrin Sponsors:   Washington University School of Medicine;   Johns Hopkins University;   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - March 20, 2018 Category: Research Source Type: clinical trials