Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101

Trial Profile

Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101

Completed
Phase of Trial: Phase I

Latest Information Update: 09 Apr 2018

At a glance

  • Drugs AVXS 101 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions
  • Sponsors AveXis
  • Most Recent Events

    • 09 Apr 2018 According to the Novartis media release, two-year data from this study will be presented at the American Academy of Neurology 2018.
    • 09 Apr 2018 Results presented in the Novartis media release.
    • 27 Mar 2018 According to an AveXis media release, based on the results from this trial, the Japan's Ministry of Health, Labour and Welfare (MHLW) has awarded SAKIGAKE Designation (SAKIGAKE) for AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1.
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