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A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension

Trial Profile

A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension

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

Latest Information Update: 25 Jun 2025

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At a glance

  • Drugs Taldefgrobep alfa (Primary)
  • Indications Spinal muscular atrophy
  • Focus Registrational; Therapeutic Use
  • Acronyms RESILIENT
  • Sponsors Biohaven Pharmaceuticals

Most Recent Events

  • 18 Jun 2025 Planned End Date changed from 1 Jan 2025 to 1 Jan 2026.
  • 25 Nov 2024 According to a Biohaven Labs media release, evaluation of additional RESILIENT clinical and biomarker data is ongoing, and the company plans to engage with FDA regarding these emerging data to discuss a path forward.Full topline data will be presented at an upcoming scientific meeting.
  • 25 Nov 2024 Results presented in a Biohaven Labs Media Release.

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