Research programme: Charcot-Marie-Tooth disease therapeutics - Charcot-Marie-Tooth Association/Sanofi Genzyme
Latest Information Update: 16 Dec 2016
At a glance
- Originator Charcot-Marie-Tooth Association; Genzyme Corporation
- Developer Charcot-Marie-Tooth Association; Sanofi Genzyme
- Class Small molecules
- Mechanism of Action Gene expression 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 Charcot-Marie-Tooth disease
Most Recent Events
- 22 Sep 2014 Early research in Charcot-Marie-Tooth disease in USA (unspecified route)