Drug Profile
Research programme: therapeutics for MECP2-associated disorders - Horizon Therapeutics plc
Alternative Names: Cysteamine and related compounds - Horizon Therapeutics plc/Technology Transfer Accelerator of South Eastern France (SATT Sud Est); Mercaptamine and related compounds for Rett syndrome and MECP2-associated disorders - Horizon Therapeutics plcLatest Information Update: 06 Nov 2023
Price :
$50
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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
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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 No
Highest Development Phases
- No development reported Rett syndrome
Most Recent Events
- 02 May 2019 Horizon Pharma is now called Horizon Therapeutics plc
- 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)