Research programme: inherited muscular disorder therapeutics - Retrophin

Drug Profile

Research programme: inherited muscular disorder therapeutics - Retrophin

Alternative Names: RE-001; RE-002; RE-003

Latest Information Update: 04 Nov 2017

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At a glance

  • Originator Retrophin
  • Developer National Center for Advancing Translational Sciences; Retrophin
  • Class Recombinant fusion proteins; Small molecules
  • Mechanism of Action Dystrophin stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity No

Highest Development Phases

  • Research Inborn genetic disorders
  • No development reported Congenital structural myopathies; Duchenne muscular dystrophy; Spinal muscular atrophy

Most Recent Events

  • 04 Nov 2017 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA (Parenteral)
  • 04 Nov 2017 No recent reports of development identified for research development in Congenital structural myopathies in USA
  • 04 Nov 2017 No recent reports of development identified for research development in Spinal-muscular-atrophy in USA
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