Research programme: spinal muscular atrophy therapeutics - PTC Therapeutics

Drug Profile

Research programme: spinal muscular atrophy therapeutics - PTC Therapeutics

Latest Information Update: 30 Apr 2018

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At a glance

  • Originator PTC Therapeutics
  • Developer PTC Therapeutics; Roche; Spinal Muscular Atrophy Foundation
  • Class Small molecules
  • Mechanism of Action Survival of motor neuron 1 protein stimulants; Survival of motor neuron 2 protein stimulants
  • 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

  • Preclinical Spinal muscular atrophy

Most Recent Events

  • 30 Apr 2018 Preclinical development is ongoing in USA (PTC Therapeutics communication/pipeline, April 2018)
  • 04 Nov 2017 No recent reports of development identified for preclinical development in Spinal-muscular-atrophy in USA (PO)
  • 18 Aug 2014 Pharmacodynamics data from in vitro and preclinical studies in Spinal muscular atrophy released by PTC Therapeutics and Roche
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