Research programme: Lysosomal storage disorder therapeutics - Amicus Therapeutics
Latest Information Update: 28 Oct 2022
At a glance
- Originator Nationwide Children's Hospital; Ohio State University
- Developer Amicus Therapeutics
- Class
- Mechanism of Action Gene transference
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- No development reported Lysosomal storage diseases
Most Recent Events
- 28 Oct 2022 No recent reports of development identified for research development in Lysosomal storage diseases in USA (Intrathecal)
- 20 Sep 2018 Celenex has been acquired and merged into Amicus Therapeutics
- 20 Sep 2018 Early research in Lysosomal storage diseases in USA (Intrathecal) prior to September 2018 (Amicus Therapeutics pipeline)