8:00 Chair's Opening Remarks
Predicting Future Success for Neuromuscular Drug Development
8.15 Assessing the Current Clinical Landscape for Neuromuscular Candidates – Is the Future Bright?
Nicolas Currier, Associate Medical Director, Biogen
8.45 Treating Today’s Patients: Moving to Restore Muscle Function in Addition to Slowing Progression
Kenneth Attie, Vice President, Medical Research, Acceleron Pharma
9.15 Speed Networking
This session is the ideal opportunity to meet face-to-face with the key thought leaders working in the neuromuscular field. Specifically designed to connect you with new contacts from the most active companies in the field, the renowned Speed Networking session will be one of the most valuable hours you will spend at NMD.
10.00 Morning Refreshments
Translating into the Clinic: Essential Considerations
11.00 Effectively Integrating Neuromuscular Biomarkers into the Clinic
Laurie Conklin, Medical & Regulatory Director, ReveraGen Biopharma
Preclinical Models of Neuromuscular Diseases: What Can We Expect?
11.30 Electrophysiology Biomarkers in Preclinical Mouse Studies: Sensitive & Reliable Outcome Measures
Laurent Bogdanik, Associate Director, Neurobiology, In Vivo Pharmacology, The Jackson Laboratory
12.00 Lunch & Networking
1.00 Limitations of Preclinical Models: Lessons Learned from Myostatin+ Targeted Therapies for NMD
Rajasekhar Suragani, Director of Preclinical & Translational, Exploratory Biology Acceleron Pharma
1.30 Mastermind: Discussing the Limited Availability & Suitability of Preclinical Models for Rare Diseases & Future Needs
- What preclinical models are currently available and how well do they represent neuromuscular diseases?
- What needs to improve for better predictability of clinical efficacy?
- Are model providers investing enough into developing rare disease models?
2.00 Afternoon Refreshments & Poster Session
The learning and networking continues at the Poster Session, an informal part of the conference agenda, allowing you to connect with your peers in a relaxed atmosphere and continue to forge new, beneficial relationships. You will have the opportunity to present and review presentations displaying new data in neuromuscular research, such as novel targets and advances in clinical development.
Navigating Clinical Trial Design Complications for Neuromuscular Disorders
3.00 How to Create Exclusion Criteria & Characterize Patient Populations Whilst Maintaining Patient Enrolment Quantity Through Phases
Ralph Kern, Executive Vice President, Chief Operating Officer & Chief Medical Officer, Brainstorm Cell Therapeutics
3.30 Accelerating Drug Development for Rare Diseases: Development of a Clinical Trial Simulation Tool for DMD
Jane Larkindale, Executive Director, Duchenne Regulatory Science Consortium, Critical Path Institute
4:00 Chair's Closing Remarks