Drug Profile
Research programme: lysosomal storage disorder therapeutics - Calporta
Latest Information Update: 28 Jul 2019
Price :
$50
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At a glance
- Originator University of Michigan
- Developer Calporta Therapeutics; University of Michigan
- Class Small molecules
- Mechanism of Action MCOLN1 protein modulators
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- No development reported Niemann-Pick disease type C
Most Recent Events
- 28 Jul 2019 No recent reports of development identified for research development in Niemann-Pick-disease-type-C in USA
- 15 Jun 2015 Early research in Niemann-Pick disease type C in USA (unspecified route)