Research programme: therapeutics for MECP2-associated disorders - Horizon PharmaAlternative Names: Cysteamine and related compounds - Horizon Pharma/Technology Transfer Accelerator of South Eastern France (SATT Sud Est); Mercaptamine and related compounds for Rett syndrome and MECP2-associated disorders - Horizon Pharma
Latest Information Update: 11 Nov 2016
At a glance
- Originator Aix-Marseille University; Curie Institute; INSERM
- Class Mercaptoethylamines; Small molecules
- Mechanism of Action Brain-derived neurotrophic factor expression stimulants; Glutathione synthase 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 No
Highest Development Phases
- No development reported Rett syndrome
Most Recent Events
- 16 Jul 2016 No recent reports of development identified for preclinical development in Rett syndrome in France
- 07 May 2013 Preclinical trials in Rett syndrome in France (unspecified route)