Validate Clinically Meaningful Endpoints, Optimize Clinical Trial Design & Compare & Contrast Diverse Approaches to Accelerate the Discovery & Development of Meaningful Treatments for Patients in Need

The 3rd Neuromuscular Drug Development  Summit presents a unique opportunity to:

• Uncover the right outcome measures to use in your clinical trial and discover how to establish clinical meaningfulness with Biogen
• Hear approaches to optimizing therapeutic efficacy in SMA, an innovative approach to trial design for real world SMA population and how combinatorial treatments may improve SMA treatment outcomes with Roche, PTC Therapeutics and John Hopkins University School of Medicine
• Assess emerging neurophysiological methods for more quantitative, faster readouts and the need to explore digital capture tools to improve therapeutic R&D with Harvard Medical School and the Parent Project Muscular Dystrophy (PPMD)
• Dissect various dosing strategies and discuss how to demonstrate a clear mechanistic route between dose, mechanisms of action and endpoints with NMD Pharma and Argenx
• Evaluate the role of biomarkers, how regulatory agencies approach the integration of biomarkers in clinical trials and biomarker outcome measures outside dystrophin with ReveraGen Biopharma and the Critical Path Institut
• Hear how Avidity Biosciences are developing an oligonucleotide antibody conjugate for the treatment of DM1

Uniting drug developers and academics from all corners of the world, the virtual Neuromuscular Drug Development Summit is your opportunity to discuss recent advances in this fast-evolving field, convey lessons learned and network to build long lasting partnerships.