Research programme: facioscapulohumeral muscular dystrophy therapeutics - Saint Louis University/Ultragenyx
Latest Information Update: 31 Mar 2016
At a glance
- Originator Saint Louis University
- Developer Saint Louis University; Ultragenyx Pharmaceutical
- Class Small molecules
- Mechanism of Action DUX4 protein inhibitors; Gene modulators
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 Facioscapulohumeral muscular dystrophy