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Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion

Trial Profile

Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion

Not yet recruiting
Phase of Trial: Phase III

Latest Information Update: 15 Feb 2019

At a glance

  • Drugs Onasemnogene-abeparvovec (Primary)
  • Indications Spinal muscular atrophy
  • Focus Therapeutic Use
  • Acronyms REACH
  • Sponsors AveXis
  • Most Recent Events

    • 08 Feb 2019 Status changed from planning to not yet recruiting.
    • 18 Jan 2018 New trial record
    • 16 Jan 2018 According to a AveXis media release, this trial may expect to start in late Q4 2018 or early 2019.
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