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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