An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Trial Profile

An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 05 Jul 2017

At a glance

  • Drugs Nusinersen (Primary)
  • Indications Spinal muscular atrophy
  • Focus Therapeutic Use
  • Acronyms NURTURE
  • Sponsors Biogen
  • Most Recent Events

    • 09 May 2017 According to a Ionis Pharmaceuticals media release, data from the study was presented at the American Academy of Neurology (AAN) annual meeting.
    • 28 Apr 2017 Interim efficacy and safety results (31 Oct 2016; n=20) presented at the 69th Annual Meeting of the American Academy of Neurology
    • 24 Apr 2017 Interim results (n=20) published in a Biogen media release.
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