Speakers
Katherine Meilleur
Principal Clinical Scientist
Neuromuscular Development Unit, Biogen
Day One
Wednesday, 2 December 2020
10:50 am | Live Q&A
9:50 am | Uncovering the Right Clinical Outcome Measures for Each Specific Neuromuscular Disease
Eric Hoffman
Associate Dean of Research/Co-Founder & VP, ReveraGen Biopharma
SUNY Binghamton School of Pharmacy
Day One
Wednesday, 2 December 2020
12:50 pm | Live Q&A
12:30 pm | Evaluating the Role of Biomarkers - Integrating Biomarkers in Clinical Trials Through DMD Case Studies
Terry Partridge
Professor of Integrative systematic Biology
Children’s National Medical Center & George Washington University
Josie Godfrey
Strategic Director
Project HERCULES
Day Two
Thursday, 3 December 2020
3:00 pm | Live Q&A
1:40 pm | Spearheading a Collaborative Effort to Drive Therapeutic Success for Patients in Need
Pat Furlong
Founding President & CEO
Parent Project Muscular Dystrophy (PPMD)
Day One
Wednesday, 2 December 2020
10:50 am | Live Q&A
10:30 am | The Need to Explore Digital/Video Capture Tools to Improve Therapeutic R&D in DMD Trials
Eva Chin
Chief Development Officer
NMD Pharma
Day One
Wednesday, 2 December 2020
4:50 pm | Live Q&A
3:40 pm | Exploring Pharmacodynamic Safety Profiles for Successful Clinical Translation
Stojan Peric
Assistant Professor, Neurologist, Neurology Clinic,
Clinical Centre of Serbia, Faculty of Medicine, University of Belgrade, Serbia
Nikolai Naryshkin
Vice President
External Research & Alliance Management, Translational Medicine, PTC Therapeutics, Inc
Day One
Wednesday, 2 December 2020
2:50 pm | Live Q&A
2:30 pm | Translational Approaches for Treatment of Neuromuscular Disorders
Maria Judit Molnarm
Professor of Neurology, Psychiatry & Clinical Genetics, Director of the Institute of Genomic Medicine & Rare Disorders,
Semmelweis University
Day Two
Thursday, 3 December 2020
9:10 am | Panel Discussion - Biggest Learnings From Failed or Abandoned Clinical Trials
Jodi Wolff
Head of Patient Advocacy
Santhera Pharmaceuticals
Day One
Wednesday, 2 December 2020
4:50 pm | Live Q&A
4:20 pm | Build It & They Will Come, Sometimes: The Burden of Trial Recruitment in Neuromuscular Disease
Charlotte J. Sumner
Professor of Neurology, Neuroscience
Johns Hopkins University School of Medicine
Day One
Wednesday, 2 December 2020
2:50 pm | Live Q&A
2:10 pm | The Challenge of Optimizing Therapeutic Efficacy in SMA
Jane Larkindale
Executive Director, Duchenne Regulatory Science Consortium
Executive Director, Rare Disease Cures Accelerator- Data and Analytics Platform
Day One
Wednesday, 2 December 2020
12:50 pm | Live Q&A
12:10 pm | Leveraging Analytical Tools & Models for More Effective Neuromuscular Drug Development
Kathyrn Wagner
Director of the Center for Genetic Muscle Disorders, Kennedy Krieger Institute, Professor of Neurology & Neuroscience, Johns Hopkins School of Medicine
Day Two
Thursday, 3 December 2020
12:30 pm | Live Q&A
12:10 pm | Exploring the Elusive Promise of Myostatin Inhibition for Muscular Dystrophies
9:10 am | Panel Discussion - Biggest Learnings From Failed or Abandoned Clinical Trials
Ranjan Batra
Vice President of Research and Development
Locana
Day Two
Thursday, 3 December 2020
12:30 pm | Live Q&A
11:50 am | Leveraging RNA Targeting CRISPR Therapeutics to Target Myotonic Dystrophy
Andrew A Wolff
Senior Fellow, Clinical Research & Development
Cytokinetics
Day One
Wednesday, 2 December 2020
9:10 am | Kick-Off Panel Discussion: Dissecting Fundamental Questions Arising for Each Therapeutic Approach
Day Two
Thursday, 3 December 2020
12:30 pm | Live Q&A
11:30 am | Clinical Development of Fast Skeletal Muscle Troponin Activators for the Potential Treatment of Neuromuscular Diseases
Mark Diamond
Managing Director & CEO
Antisense Therapeutics
Day Two
Thursday, 3 December 2020
10:30 am | Live Q&A
9:50 am | Developing an Antisense Inhibitor of the CD49d Receptor for DMD Patients
Art Levin
CSO
Avidity Biosciences
Day Two
Thursday, 3 December 2020
10:30 am | Live Q&A
10:10 am | Pioneering a New Class of Oligonucleotide Therapies: Developing an Oligonucleotide Antibody Conjugate for the Treatment of DM1
Seward Rutkove
Professor of Neurology & Chief of the Division of Neuromuscular Disease
Harvard Medical School/Beth Israel Deaconess Medical Center
Day One
Wednesday, 2 December 2020
10:50 am | Live Q&A
10:10 am | Emerging Neurophysiological Methods – New Assessment Tools
Peter Ulrichts
Scientific Lead Neuromuscular Franchise
Argenx
Day One
Wednesday, 2 December 2020
4:50 pm | Live Q&A
4:00 pm | Phase 3 Myasthenia Gravis Study of the FcRn Antagonist Efgartigimod
Paulo Fontoura
Global Head & SVP, Neuroscience & Rare Diseases Clinical Development
Roche
Day One
Wednesday, 2 December 2020
2:50 pm | Live Q&A
1:50 pm | Discovering an Innovative Approach to Trial Design for Real World SMA Population
9:10 am | Kick-Off Panel Discussion: Dissecting Fundamental Questions Arising for Each Therapeutic Approach
Kristina Sjöblom Nygren
CMO, Head of Development
Santhera Pharmaceuticals
Day Two
Thursday, 3 December 2020