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A Prospective, Multicenter, Multinational, Observational Study of Onasemnogene Abeparvovec-xioi Assessing Outcomes and Treatment Patterns in Patients with Spinal Muscular Atrophy Through an Integrated Restore Registry

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Trial Profile

A Prospective, Multicenter, Multinational, Observational Study of Onasemnogene Abeparvovec-xioi Assessing Outcomes and Treatment Patterns in Patients with Spinal Muscular Atrophy Through an Integrated Restore Registry

Status: Recruiting
Phase of Trial: Phase IV

Latest Information Update: 20 Jun 2023

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At a glance

  • Drugs Onasemnogene-abeparvovec (Primary) ; Nusinersen; Risdiplam
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms RESTORE
  • Sponsors Novartis Gene Therapies

Most Recent Events

  • 01 Jun 2023 Results (at data cut off May 23, 2022), evaluating baseline characteristics, and post-treatment motor function, motor milestone achievement, use of ventilatory/nutritional support, and adverse events , published in the Pediatric Dermatology
  • 19 Mar 2023 According to a Novartis media release, results of this study being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.
  • 26 Apr 2022 Results (as of data cut off, n=247) presented at the 74th Annual Meeting of the American Academy of Neurology 2022

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