Research programme: rare genetic disorder therapeutics - Massachusetts General Hospital/PTC Therapeutics
Latest Information Update: 18 Dec 2015
At a glance
- Originator Massachusetts General Hospital
- Developer PTC Therapeutics
- Class Small molecules
- Mechanism of Action Undefined mechanism
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
On Fast track
Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.
- New Molecular Entity Yes
Highest Development Phases
- Research Inborn genetic disorders