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: 07 Sep 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.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy
  • Research Congenital structural myopathies; Inborn genetic disorders; Spinal muscular atrophy

Most Recent Events

  • 05 Sep 2017 Retrophin enters into a Co-operative Research and Development agreement with National Center for Advancing Translational Sciences and for the development of NGLY1 deficiency therapeutics
  • 05 Sep 2017 Early research in Inborn genetic disorders in USA (unspecified route)
  • 28 Nov 2014 Active development for Duchenne muscular dystrophy is ongoing in USA
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