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A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101

Trial Profile

A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101

Recruiting
Phase of Trial: Phase I

Latest Information Update: 03 May 2018

At a glance

  • Drugs Onasemnogene-abeparvovec (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions
  • Sponsors AveXis
  • Most Recent Events

    • 31 Aug 2018 Biomarkers information updated
    • 03 May 2018 Results published in the Media Release
    • 24 Apr 2018 According to an AveXis media release, two-year data from this study will be presented at the American Academy of Neurology 2018.
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