Moving Beyond Variable & Subjective Muscle Function Assessments: Selecting Endpoints with Clinical Approval in Mind
10:00 am - Tuesday 26th January 2027
As the landscape of therapeutic approaches for muscular disorders expands, so must the endpoints used to assess them. Demonstrating meaningful clinical benefit remains one of the biggest barriers to successful approval, and while functional assessments continue to play an important role, sponsors are exploring novel biomarkers, digital measures and disease-specific endpoints that can provide earlier, more sensitive evidence of treatment effect. Attendees will gain practical insights into endpoint selection, validation and implementation from experts navigating today’s evolving regulatory landscape.
This workshop will:
- Explore the shortfalls of traditional functional assessments such as the 6-minute walk test and the North Star Ambulatory Assessment to identify the key challenges like variability in a largely heterogeneous patient population
- Discuss aligning endpoint selection with disease biology to demonstrate clinically meaningful benefits
- Discuss novel strategies including digital endpoints to detect treatment effects earlier and generate evidence that supports regulatory decision-making
