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A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101

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A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101

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

Latest Information Update: 05 Mar 2024

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At a glance

  • Drugs Onasemnogene-abeparvovec (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions
  • Acronyms START LTFU
  • Sponsors Novartis Gene Therapies; Novartis Pharmaceuticals

    • Most Recent Events

    • 27 Feb 2024 Planned End Date changed from 1 Dec 2033 to 2 Dec 2030.
    • 27 Feb 2024 Planned primary completion date changed from 1 Dec 2033 to 2 Dec 2030.
    • 27 Apr 2023 Results (As of May 23, 2022, n=13 ) presented at the 75th Annual Meeting of the American Academy of Neurology 2023

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