Research programme: small molecules therapeutics - GABAeron
Latest Information Update: 10 Feb 2022
At a glance
- Originator GABAeron
- Class Small molecules
- Mechanism of Action Sterol modulators
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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
- Preclinical Smith-Lemli-Opitz syndrome
Most Recent Events
- 01 Feb 2022 Preclinical trials in Smith-Lemli-Opitz syndrome in USA before February 2022 (GABAeron pipeline, February 2022)