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Research programme: spinocerebellar ataxia therapeutics - PTC Therapeutics

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Drug Profile

Research programme: spinocerebellar ataxia therapeutics - PTC Therapeutics

Latest Information Update: 18 Feb 2024

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At a glance

  • Originator PTC Therapeutics
  • Class Neuroprotectants; Small molecules
  • Mechanism of Action RNA splicing modulators
  • 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
  • Available For Licensing Yes

Highest Development Phases

  • Research Machado-Joseph Disease; Spinocerebellar ataxias

Most Recent Events

  • 18 Feb 2024 Early research in Machado-Joseph Disease in USA (PO) (PTC Therapeutics pipeline January 2024)
  • 11 Dec 2021 Research programme: spinocerebellar ataxia therapeutics - PTC Therapeutics is available for licensing as of 11 Dec 2021. https://www.ptcbio.com/our-science/expertise/
  • 01 Dec 2021 Early research in Spinocerebellar ataxias in USA (PO) before December 2021 (PTC Therapeutics pipeline, December 2021)

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