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Expert Opin Drug Discov. 2019 May;14(5):499-509. doi: 10.1080/17460441.2019.1588882. Epub 2019 Mar 19.

Animal models for Niemann-Pick type C: implications for drug discovery & development.

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a Orphazyme A/S , Copenhagen , Denmark.


Niemann-Pick type C (NPC) is a neurovisceral, progressively detrimental lysosomal storage disease with very limited therapeutic options and no approved treatment available in the US. Despite its rarity, NPC has seen increased drug developmental efforts over the past decade, culminating in the completion of two potential registration trials in 2018. Areas covered: This review highlights the many available animal models that have been developed in the field and briefly covers classical and new cell technologies. This review provides a high-level evaluation and prioritization of the various models with regard to efficient and clinically translatable drug development, and briefly discusses the relevant developments and opportunities pertaining to this. Expert opinion: With a number of in vitro and in vivo models available, and with having several drugs, all with various mechanisms of action, either approved or in late stage development, the NPC field is in an exciting time. One of the challenges for researchers and developers will be the ability to make use of the lessons learnt from existing late-stage programs as well as the incorporation not only of the opportunities but also the limitations of the many models into successful drug discovery and translational development programs.


Animal model; NPC; NPC1; NPC2; Niemann-Pick type C; cholesterol; drug development; glycosphingolipids; lysosomal storage diseases; lysosomes; sphingolipids

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