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Long-Term Follow-Up of A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

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Trial Profile

Long-Term Follow-Up of A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Status: Active, no longer recruiting
Phase of Trial: Phase IV

Latest Information Update: 03 Oct 2024

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At a glance

  • Drugs Cobnabexagene anvuparvovec (Primary)
  • Indications Sandhoff disease; Tay-Sachs disease
  • Focus Adverse reactions
  • Most Recent Events

    • 03 Oct 2024 New trial record

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