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Research programme: spinal muscular atrophy therapeutics - PTC Therapeutics

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Drug Profile

Research programme: spinal muscular atrophy therapeutics - PTC Therapeutics

Alternative Names: SMN-C2; SMN-C3

Latest Information Update: 28 Apr 2025

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At a glance

  • Originator PTC Therapeutics
  • Developer PTC Therapeutics; Roche
  • 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.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • No development reported Neuromuscular disorders; Spinal muscular atrophy

Most Recent Events

  • 28 Apr 2025 No recent reports of development identified for research development in Neuromuscular-disorders in USA
  • 28 Dec 2021 No recent reports of development identified for preclinical development in Spinal-muscular-atrophy in USA (PO)
  • 10 Mar 2021 Early research in Neuromuscular disorders in USA (unspecified route)

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