Research programme: protein modulators - PTC Therapeutics

Drug Profile

Research programme: protein modulators - PTC Therapeutics

Alternative Names: DBMD-Utrophin; DMD Exon-51; Project Catalyst; SERCA2a protein stimulants - PTC Therapeutics

Latest Information Update: 28 Jan 2014

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At a glance

  • Originator PTC Therapeutics
  • Class Small molecules
  • Mechanism of Action Dystrophin expression modulators; Insulin-like growth factor I stimulants; Myostatin inhibitors; Sarcoplasmic reticulum calcium-transporting ATPase stimulants; Utrophin activators
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Research Duchenne muscular dystrophy

Most Recent Events

  • 28 Jan 2014 Early research for Duchenne muscular dystrophy is ongoing in USA (PO)
  • 15 Dec 2011 This programme is still in active development
  • 31 Dec 2003 Early research in Duchenne muscular dystrophy in USA (unspecified route)
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