Drug Profile
Research programme: spinal muscular atrophy therapeutics - PTC Therapeutics
Alternative Names: SMN-C2; SMN-C3Latest Information Update: 28 Dec 2021
Price :
$50
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At a glance
- Originator PTC Therapeutics
- Developer PTC Therapeutics; Roche
- Class Small molecules
- Mechanism of Action Survival of motor neuron 1 protein stimulants; Survival of motor neuron 2 protein stimulants
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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
- Research Neuromuscular disorders
- No development reported Spinal muscular atrophy
Most Recent Events
- 28 Dec 2021 No recent reports of development identified for preclinical development in Spinal-muscular-atrophy in USA (PO)
- 10 Mar 2021 Early research in Neuromuscular disorders in USA (unspecified route)
- 30 Apr 2018 Preclinical development is ongoing in USA (PTC Therapeutics communication/pipeline, April 2018)