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National Institutes of Health, National Center for Advancing Translational Sciences — Rare Disease Pipeline | Kisho | Kisho
National Institutes of Health, National Center for Advancing Translational Sciences
Other
1
Orphan Designations
0
FDA Approvals
1
Rare Diseases
0
News Articles
Pipeline
Designations
News
Diseases
Disease
Drug(s)
Designation
Approved
propionic acidemia
Adeno-Associated Virus 9 human Propionyl-CoA Carboxylase, alpha subunit (AAV9-hPCCA)
Orphan Designation
-
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