Research programme: neurometabolic disease therapeutics - Minoryx Therapeutics
Latest Information Update: 20 Dec 2016
At a glance
- Originator Minoryx Therapeutics
- Class Small molecules
- Mechanism of Action Glycoside hydrolase 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 Gangliosidoses; Mucopolysaccharidosis IV
Most Recent Events
- 19 Dec 2016 Early research in Mucopolysaccharidosis IV in Spain (unspecified route) before December 2016
- 19 Dec 2016 Early research in Gangliosidosis in Spain (unspecified route) before December 2016