Research programme: spinal muscular atrophy therapeutics - deCODE genetics/Families of Spinal Muscular Atrophy

Drug Profile

Research programme: spinal muscular atrophy therapeutics - deCODE genetics/Families of Spinal Muscular Atrophy

Latest Information Update: 07 Apr 2015

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At a glance

  • Originator deCODE genetics; Families of SMA
  • Class Piperidines; Quinazolines; Small molecules
  • Mechanism of Action Survival of motor neuron 1 protein stimulants
  • Orphan Drug Status

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

    Yes - Spinal muscular atrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Spinal muscular atrophy

Most Recent Events

  • 28 Aug 2009 Quinazoline 495 receives Orphan Drug status for Spinal muscular atrophy in USA
  • 15 Aug 2008 Research programme is still in preclinical trials for Spinal muscular atrophy in USA and Iceland
  • 26 Jun 2007 A clinical candidate has been selected for spinal muscular atrophy
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