Cln2 Disease

• CLINICAL_TRIALS research · 2025-10-24
  Clinical Trial Update: Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

  This update indicates an ongoing study evaluating developmental outcomes in children diagnosed with CLN2 disease (Batten disease), currently…

• CLINICAL_TRIALS research · 2025-09-12
  Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

  This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…

• CLINICAL_TRIALS trial_result · 2022-08-24
  Clinical Trial Update: An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease [Phase 1, Phase 2]

  An extension study assessed long-term efficacy and safety of BMN 190 in patients with CLN2 disease (a form of Batten disease), reporting res…

• FDA approval · 2018-12-11
  FDA Approved: BRINEURA — Supplemental Application (BioMarin Pharmaceutical Inc.)

  The FDA approved a supplemental application for BRINEURA (cerliponase alfa) from BioMarin Pharmaceutical Inc., updating the drug’s approved …

• CLINICAL_TRIALS trial_result · 2018-06-11
  Clinical Trial Update: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease [Phase 1, Phase 2]

  Phase 1/2 open-label dose-escalation trial evaluating intracerebroventricular BMN 190 for safety, tolerability, PK, and efficacy in CLN2 dis…

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• CLINICAL_TRIALS approval · 2017-07-24
  Clinical Trial Update: A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease

  Marketing approval granted for BioMarin's BMN 190 (cerliponase alfa) to treat CLN2 disease; the therapy is now approved for marketing.