Drug Profile
Research programme: rare genetic disorder therapeutics - Massachusetts General Hospital/PTC Therapeutics
Alternative Names: PTC-258Latest Information Update: 28 Jan 2020
Price :
$50
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At a glance
- Originator Massachusetts General Hospital
- Developer PTC Therapeutics
- Class Small molecules
- Mechanism of Action Undefined mechanism
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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 Huntington's disease
- Discontinued Inborn genetic disorders
Most Recent Events
- 28 Jan 2020 No recent reports of development identified for research development in Huntington's-disease in USA (PO)
- 29 Oct 2019 Discontinued - Preclinical for Inborn genetic disorders (Familial dysautonomia) in USA (PO)
- 29 Oct 2019 PTC Therapeutics plans a clinical trial for Huntington's programme in 2020