Challenges in Clinical Outcome Selection & Validation in Neuromuscular Gene Therapy

Time: 11:15 am
day: Day One


  • The impact of accelerated approvals on clinical outcome considerations
  • Defining outcomes that are likely to predict clinical benefit
  • Exploring the relationship between functional measures and clinical biomarkers
  • Navigating the heterogeneity of neuromuscular diseases and how this impacts clinical endpoints and control variables
  • Leveraging natural history data to inform study design considerations and responder definitions
  • Administrative and rater training considerations with young children with neuromuscular disorders