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 Institute
  • 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.

Expert Speaker Faculty Includes:

Katherine Meilleur

Principal Clinical Scientist

Neuromuscular Development Unit, Biogen

Eva Chin

Chief Development Officer

NMD Pharma

Ranjan Batra

Vice President of Research and Development


Peter Ulrichts

Scientific Lead Neuromuscular Franchise


Paulo Fontoura

Global Head & SVP, Neuroscience & Rare Diseases Clinical Development


"The event allowed great access to senior people and provided opportunities to collaborate and develop opportunities for further interaction. The speakers provided excellent insight into the state of play in the research areas covered and what still needs to be done. Very worthwhile."

Steven Arnott, Chief Executive Officer, Perron Institute

"Excellent networking and learning opportunity for pharma scientists." 

Matthew Kennedy, Director, Merck

"Excellent conference with a very good and diverse set of speakers and topics."

Jim Cassella, Chief Development Officer, Concert Pharmaceuticals

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