Explore the Agenda
9:00 am Morning Coffee & Workshop Check-In
Workshop A
10:00 am Moving Beyond Variable & Subjective Muscle Function Assessments: Selecting Endpoints with Clinical Approval in Mind
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
1:00 pm Lunch & Networking
Workshop B
2:00 pm Exploring Combining Modalities for Muscular Dystrophies to Enhance Efficacy Without Increasing Toxicity
As current treatments reach the limit of their therapeutic potential, combination approaches are emerging as a promising strategy to address the complexities of treating muscular disorders. Combining complementary MOAs may enhance efficacy, durability and patient outcomes while minimizing the need for higher doses, limiting toxicity and improving safety. Attendees will learn how leading developers are identifying synergistic combinations and navigating the challenges of demonstrating added benefit while maintaining acceptable safety profiles.
This workshop will:
- Combine complementary therapeutic modalities to target multiple drivers of disease and maximize treatment efficacy
- Utilize translational and clinical evidence to identify synergistic combinations and limit cross-modality interactions that lead to toxicity
- Explore combination therapy successes for neuromuscular diseases: triple drug combination for sporadic ALS