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Research programme: personalised orphan disease therapeutics - Perlara

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Drug Profile

Research programme: personalised orphan disease therapeutics - Perlara

Alternative Names: APOL1 kidney disease therapeutic - Perlara; Batten disease therapeutic - Perlara; Leigh syndrome therapeutic - Perlara; NGLY1 therapeutic - Perlara; NPC1 therapeutic - Perlara; PERL 101

Latest Information Update: 28 Jan 2023

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At a glance

  • Originator Perlstein Lab
  • Developer Perlara
  • Class Small molecules
  • Mechanism of Action Undefined mechanism
  • 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 - Niemann-Pick disease type C

Highest Development Phases

  • No development reported Inborn genetic disorders; Kidney disorders; Leigh disease; Mucopolysaccharidosis III; Neuronal ceroid lipofuscinosis; Niemann-Pick disease type C

Most Recent Events

  • 28 Jan 2023 No recent reports of development identified for research development in Kidney-disorders in USA
  • 28 Oct 2022 No recent reports of development identified for research development in Mucopolysaccharidosis-III in USA
  • 28 Feb 2020 No recent reports of development identified for research development in Inborn genetic disorders in USA

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