Speakers

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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

3:00 pm | Live Q&A

2:00 pm | Evolving Strategies to Manage a Phase III Study on Respiratory Function in Patients at Risk during COVID-19