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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

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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: Completed
Phase of Trial: Phase II

Latest Information Update: 04 Jul 2025

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At a glance

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

Most Recent Events

  • 27 Jun 2025 Results presented in the Biogen Media Release.
  • 13 Jan 2025 Planned End Date changed from 31 Dec 2024 to 17 Dec 2024.
  • 13 Jan 2025 Planned primary completion date changed from 31 Dec 2024 to 17 Dec 2024.

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