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Phase I/IIa Intrathecal Gene Delivery Clinical Trial for IGHMBP2-Related Diseases

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Trial Profile

Phase I/IIa Intrathecal Gene Delivery Clinical Trial for IGHMBP2-Related Diseases

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 12 Jan 2022

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At a glance

  • Drugs ACTX-401 (Primary)
  • Indications Charcot-Marie-Tooth disease; Respiratory distress syndrome; Spinal muscular atrophy
  • Focus Adverse reactions

    • Most Recent Events

    • 16 Dec 2021 New trial record

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