Speakers
Katherine Meilleur
Principal Clinical Scientist
Neuromuscular Development Unit, Biogen
Day One
Wednesday, 2 December 2020
11:00 am | Live Q&A
9:45 am | Outcome Measures in Neuromuscular Disease: Establishing Minimal Clinically Important Difference (MCID)
Eric Hoffman
Associate Dean of Research/Co-Founder & VP, ReveraGen Biopharma
SUNY Binghamton School of Pharmacy
Day One
Wednesday, 2 December 2020
1:05 pm | Live Q&A
12:40 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
Day Two
Thursday, 3 December 2020
9:10 am | Panel Discussion - Biggest Learnings From Failed or Abandoned Clinical Trials
Josie Godfrey
Strategic Director
Project HERCULES
Day Two
Thursday, 3 December 2020
2:55 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
11:00 am | Live Q&A
10:35 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
5:05 pm | Live Q&A
3:50 pm | Exploring Pharmacodynamic Safety Profiles for Successful Clinical Translation
Day Two
Thursday, 3 December 2020
9:10 am | Panel Discussion - Biggest Learnings From Failed or Abandoned Clinical Trials
Nikolai Naryshkin
Vice President
External Research & Alliance Management, Translational Medicine, PTC Therapeutics, Inc
Day One
Wednesday, 2 December 2020
3:05 pm | Live Q&A
2:40 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
5:05 pm | Live Q&A
4:40 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
3:05 pm | Live Q&A
2:15 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
1:05 pm | Live Q&A
12:15 pm | Leveraging Analytical Tools & Models for More Effective Neuromuscular Drug Development
9:05 am | Kick-Off Panel Discussion: Dissecting Fundamental Questions Arising for Each Therapeutic Approach
Kathryn Wagner
Vice President, Global Head of Neuromuscular Disorders, F. Hoffmann-La Roche Ltd
Day Two
Thursday, 3 December 2020
12:45 pm | Live Q&A
12:20 pm | Exploring the Elusive Promise of Myostatin Inhibition for Muscular Dystrophies
Ranjan Batra
Vice President of Research and Development
Locana
Day Two
Thursday, 3 December 2020
12:45 pm | Live Q&A
11:55 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
9:05 am | Kick-Off Panel Discussion: Dissecting Fundamental Questions Arising for Each Therapeutic Approach
Day Two
Thursday, 3 December 2020
12:45 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
10:40 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
10:40 am | Live Q&A
10:15 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
11:00 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
5:05 pm | Live Q&A
4:15 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
3:05 pm | Live Q&A
1:50 pm | Discovering an Innovative Approach to Trial Design for Real World SMA Population
9:05 am | Kick-Off Panel Discussion: Dissecting Fundamental Questions Arising for Each Therapeutic Approach
Pavel Balabanov
Head of Therapies for Neurological & Psychiatric Disorders
European Medicines Agency
Day One
Wednesday, 2 December 2020
9:05 am | Kick-Off Panel Discussion: Dissecting Fundamental Questions Arising for Each Therapeutic Approach
Sharon Hesterlee
EVP, Chief Research Officer
Muscular Dystrophy Association
Day Two
Thursday, 3 December 2020
2:30 pm | Data Collection at Point of Care – MDA MOVR Database Yields First Insights
2:55 pm | Live Q&A
Debra Miller
Founder & CEO
CureDuchenne
Day Two
Thursday, 3 December 2020
9:10 am | Panel Discussion - Biggest Learnings From Failed or Abandoned Clinical Trials
Laurent Bogdanik
Associate Director, In Vivo Pharmacology Services, Neurobiology
Jackson Laboratory
Day One
Wednesday, 2 December 2020
1:05 pm | Live Q&A
11:50 am | EMG, Force Recording or Neuromuscular Junctions Histology: Which is Best for NMD Preclinical Trials?
Sabine Pilot
Head Clinical Operations
Santhera Pharmaceuticals
Day Two
Thursday, 3 December 2020