Research programme: therapeutics for MECP2-associated disorders - Horizon Pharma

Drug Profile

Research programme: therapeutics for MECP2-associated disorders - Horizon Pharma

Alternative 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

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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
  • 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

  • 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)
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