8:00 Chair's Opening Remarks
Promising Therapeutic Approaches for Neuromuscular Disorders
8.15 Exploring Targeted Delivery of Oligonucleotide Therapeutics to Muscle
Romesh Subramanian, CEO & Founder, Dyne Therapeutics
8.45 Focused Evolution of CNS-targeting AAV Vectors Using Cell TypeSpecific RNA Expression
Mathieu Nonnenmacher, Principal Scientist, Vector Engineering, Voyager Therapeutics
9.15 Is There Still a Role for Small Molecule Treatments for Neuromuscular Disorders?
Kirsten Gruis, Global Head, Neuromuscular, Roche
9.45 Morning Refreshments & Networking
Supporting Neuromuscular Drug Development Commercial Success
11.00 Patient Advocacy Partnerships to Improve Real World Evidence (RWE) in Neuromuscular Drug Development
Shazia Ahmad, Director Patient & Physician Services, UBC
11.45 Panel Discussion: Encouraging Investment into Rare Diseases Research & Securing ROI
- Discussing the cost of drugs, access & reimbursement for rare neuromuscular disorders
- Discovering the collaborations needed for patient treatment success & stakeholders required
- What does an optimal deal structure & pricing scheme look like?
12.15 Lunch & Networking
Improving Drug Development Strategies for Treating Neuromuscular Diseases
1.15 Development of Combination Therapies for Rare Neuromuscular Diseases
Eva Chin, Chief Development Officer, NMD Pharma
1.45 Unbiased Discovery of Novel Targets for ALS and FTD
Kenneth Rhodes, Chief Scientific Officer, Yumanity Therapeutics
2.15 Afternoon Networking & Refreshments
FDA Approval – Is This Therapeutic Success?
2.45 Discussing the Future of Neuromuscular Therapeutics
Stuart Peltz, Founder & Chief Executive Officer, PTC Therapeutics
3.15 Panel Discussion: Assessment of Approval Considerations
- Is efficacy or biomarker improvement more important? (DMD vs. SMA approval)
- Aligning clinical expectations with current regulation – what needs to change?
- Supporting clinical scales favoured by clinicians but not recognised by regulation
3:55 Chair's Closing Remarks