Speakers

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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

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

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
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

Sabine Pilot
Head Clinical Operations
Santhera Pharmaceuticals

Day Two

Thursday, 3 December 2020

2:55 pm | Live Q&A

2:05 pm | The Operational Challenges of Conducting an Interim Analysis During COVID-19