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Science + Therapies Pipeline
A short summary of the research and clinical trials being conducted that impact Niemann-Pick disease.
Program
Current Development Phase
Milestone(s)
Discovery
Preclinical
Phase 1
Phase 2
Phase 3
mRNA gene therapy
The Florey Research Institute of Neuroscience and Mental Health ↪️
Discovery Stage
Current aims include: Investigate potential mRNA therapeutic candidates…
RAINBOW
Azafaros ↪️
Phase 2 Study
RAndomised, double-blINd, placeBo-controlled, multicenter, 12-week study to…
Arimoclomol
Zevra Therapeutics ↪️
NDA Submission
Oraly-delivered, first-in-class investigational product candidate for NPC.
Arimoclomol…
If you would like to submit your program or have updated information, please contact the Australian NPC Disease Foundation at info@npcd.org.au.
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