Research programme: spinal muscular atrophy therapeutics - PTC Therapeutics
Latest Information Update: 18 Aug 2014
At a glance
- Originator PTC Therapeutics
- Developer PTC Therapeutics; Roche; Spinal Muscular Atrophy Foundation
- Class Small molecules
- Mechanism of Action Survival of motor neuron 1 protein stimulants; Survival of motor neuron 2 protein stimulants
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
- Preclinical Spinal muscular atrophy