Drug Profile

SMN C1

Alternative Names: SMN-C1

Latest Information Update: 28 Jul 2021

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At a glance

Originator PTC Therapeutics
Developer PTC Therapeutics; Roche
Class Imidazoles; Piperazines; Pyrazines; Small molecules
Mechanism of Action 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.

No
New Molecular Entity Yes

Highest Development Phases

No development reported Spinal muscular atrophy

Most Recent Events

28 Jul 2021 No recent reports of development identified for preclinical development in Spinal-muscular-atrophy in USA (PO)
04 Jul 2019 Preclinical trials in Spinal muscular atrophy in USA (PO)
28 Jun 2019 Pharmacodynamics data from preclinical studies in Spinal muscular atrophy released by Cytokinetics

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