PRO 053

Drug Profile

PRO 053

Alternative Names: BMN-053; PRO053; PS-524

Latest Information Update: 10 Sep 2017

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At a glance

  • Originator Prosensa
  • Developer BioMarin Nederland
  • Class Antisense oligonucleotides
  • Mechanism of Action Antisense RNA modulators; Dystrophin expression stimulants
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Duchenne muscular dystrophy

Most Recent Events

  • 31 May 2016 Discontinued - Phase-II for Duchenne muscular dystrophy in France, Belgium, United Kingdom, Netherlands, Italy (IV)
  • 31 May 2016 Discontinued - Phase-II for Duchenne muscular dystrophy in France, Belgium, United Kingdom, Netherlands, Italy (SC)
  • 28 Apr 2015 Prosensa Therapeutics is now called BioMarin Nederland
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