Challenges in Clinical Outcome Selection & Validation in Neuromuscular Gene Therapy
Time: 11:15 am
day: Day One
Details:
- 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
