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A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease

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Trial Profile

A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 24 Feb 2025

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At a glance

  • Drugs Cerliponase alfa (Primary)
  • Indications Neuronal ceroid lipofuscinosis
  • Focus Therapeutic Use
  • Sponsors BioMarin Pharmaceutical

    • Most Recent Events

    • 24 Jul 2024 Results published in a BioMarin Pharmaceutical Inc media release
    • 24 Jul 2024 According to BioMarin Pharmaceutical Inc media release, company today announced that US FDA has approved supplemental Biologics License Application (sBLA) for BRINEURA (cerliponase alfa) to slow loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 deficiency.
    • 23 Jul 2024 According to BioMarin Pharmaceutical Inc media release, results from this study were presented at the 20th Annual at Organizing Research on Lysosomal Diseases meeting (WORLDSymposium) in Feb.

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