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An Open-Label, Non-Comparative Clinical Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene (ANB-004 (JSC BIOCAD, Russia)) After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy

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Trial Profile

An Open-Label, Non-Comparative Clinical Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene (ANB-004 (JSC BIOCAD, Russia)) After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 23 Feb 2024

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At a glance

  • Drugs ANB-004 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms BLUEBELL
  • Sponsors Biocad

Most Recent Events

  • 07 Mar 2023 New trial record

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