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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

Status: Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 29 Sep 2023

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At a glance

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

    • 01 Aug 2023 Results (data cut: February 15, 2021, additional 2 y of follow-up) assessing benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy published in the Muscle and Nerve
    • 14 Mar 2022 Results published in the Biogen Media Release.
    • 14 Mar 2022 According to a Biogen media release, latest results from the study will be presented at the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
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