Research programme: facioscapulohumeral muscular dystrophy therapeutics - Saint Louis University/Ultragenyx
Latest Information Update: 28 Jul 2018
At a glance
- Originator Saint Louis University
- Developer Saint Louis University; Ultragenyx Pharmaceutical
- Class Small molecules
- Mechanism of Action DUX4 protein inhibitors; Gene 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 Facioscapulohumeral muscular dystrophy