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A 12-month prospective study assessing change in respiratory function in pediatric spinal muscular atrophy during first year of nusinersen treatment and compare to previous function

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Trial Profile

A 12-month prospective study assessing change in respiratory function in pediatric spinal muscular atrophy during first year of nusinersen treatment and compare to previous function

Status: Completed
Phase of Trial: Phase IV

Latest Information Update: 12 Jul 2023

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At a glance

  • Drugs Nusinersen (Primary)
  • Indications Spinal muscular atrophy
  • Focus Pharmacodynamics

Most Recent Events

  • 24 May 2023 Results assessing the respiratory muscle effects (3 Year Follow-up) of nusinersen are maintained and describe the longer-term trajectory in lung function and Apnoea-Hypopnoea Indices in children with nusinersen treated Spinal Muscular Atrophy, presented at the 119th International Conference of the American Thoracic Society.
  • 07 May 2021 Results published in the Thorax
  • 03 Jul 2020 New trial record

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