Research programme: facioscapulohumeral muscular dystrophy therapeutics - Saint Louis University/Ultragenyx

Drug Profile

Research programme: facioscapulohumeral muscular dystrophy therapeutics - Saint Louis University/Ultragenyx

Latest Information Update: 28 Jul 2018

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At a glance

  • Originator Saint Louis University
  • Developer Saint Louis University; Ultragenyx Pharmaceutical
  • Class Small molecules
  • Mechanism of Action DUX4 protein inhibitors; Gene modulators
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes

Highest Development Phases

  • No development reported Facioscapulohumeral muscular dystrophy

Most Recent Events

  • 28 Jul 2018 No recent reports of development identified for research development in Facioscapulohumeral-muscular-dystrophy in USA
  • 23 Mar 2016 Saint Louis University and Ultragenyx Pharmaceutical collaborate for Facioscapulohumeral muscular dystrophy therapeutics in USA
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