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: 02 Nov 2017

At a glance

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

    • 02 Nov 2017 Results assessing outcomes of single dose gene-replacement therapy for spinal muscular atrophy (data cut off: August, 2017), published in the New England Journal of Medicine
    • 01 Nov 2017 Results published in an AveXis media release.
    • 03 Oct 2017 Top-line data as of August 7, 2017 will be presented at the 22nd International Annual Congress of the World Muscle Society (WMS), according to an AveXis media release. A manuscript regarding these data has been accepted for publication in a major medical journal.
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