Opening Lecture

Brendan Frey
Keynote Speaker

Professor at the University of Toronto,
Founder, CEO and Chief Engineer at Deep Genomics, Canada

Brendan Frey is an acclaimed entrepreneur, engineer and scientist. He is Founder, CEO and Chief Engineer of Deep Genomics and as far back as 2004, he began laying the foundations of deep learning for drug discovery. In 2019, Frey’s work led to the first therapeutic candidate discovered by artificial intelligence.

Why AI will be the Foundation of 21st Century Medicine

By driving cars, beating humans at their own games, transcribing speech and translating text, new AI technologies are changing the world. Brendan Frey, a world-leading expert in AI and medicine, will explain how the recent exponential growth in biomedical data and a new generation of AI technology is creating a new foundation that will propel medicine forward for the next century. Already, AI can predict how genetic variation will alter the cellular determinants of disease, which mechanisms can counteract these cellular determinants, and which molecules will be beneficial. Trends indicate that this disruption is happening more rapidly than anticipated and creating unique challenges, such as establishing trust with regulators and clinicians. Frey paints a picture of a future in which AI will profoundly alter society for hundreds of years.

Debate 1

Pre-Clinical Research - Are large animal models necessary in translational research?

Alan Beggs

Boston Children's Hospital,
Harvard Medical School,


Caroline Le Guiner
Debate Speaker

Translational Gene Therapy Laboratory, Université De Nantes, Inserm UMR 1089, CHU De Nantes, France

Caroline Le Guiner has over 17 years of experience in translational research in rAAV-based gene therapy for rare diseases, especially for the treatment of muscular diseases, including Duchenne Muscular Dystrophy. Her role is to implement gene therapy strategies from proof of concept phases until the beginning of Phase I/II clinical trials.

Annemieke Aartsma-Rus
Debate Speaker

Leiden University Medical
Center, Netherlands

Annemieke Aartsma-Rus is a professor of translational genetics at the Leiden University Medical Center. She has focused most of her career on the preclinical development of antisense-oligonucleotide mediated exon skipping for Duchenne muscular dystrophy. She is member of the executive committee of TREAT-NMD and President of the Oligonucleotide Therapeutics Society.

Debate 2

Man, or machine? Who should evaluate patients in clinical trials?

Jean-Yves Hogrel

Institute of Myology,

Jean-Yves Hogrel is the Director of the Neuromuscular Physiology and Evaluation Laboratory (Neuromuscular Investigation Center, Institute of Myology, Paris, France). His research interests mainly focus on the quantified evaluation of the neuromuscular function, particularly during therapeutic trials. His laboratory is now recognized as one of the leading laboratories in outcome measures and in innovative technologies dedicated to neuromuscular function assessment.

Lindsay Alfano
Debate Speaker

The Abigail Wexner
Research Institute at
Nationwide Children's
Hospital, USA

Lindsay Alfano is a physical therapist with expertise in the care and evaluation of patients with neuromuscular disease including outcome measure development, evaluation, and administration. Her research goals focus on standardizing training in rare disease, as well as developing optimal outcomes to measure change in abilities while minimizing testing burden.

Laurent Servais
Debate Speaker

University of Oxford,
United Kingdom

Laurent Servais is Professor of Paediatrics Neuromuscular Diseases at the University of Oxford, UK and of Liege, Belgium. He is passionate about clinical research, outcome measures, new-born screening and patients care.

Debate 3

Should neuromuscular diseases be reduced through pre-conception carrier testing?

Nathalie Goemans

Univeristy Hospitals
Leuven, Belgium

Nathalie Goemans, M.D, PhD, is a child neurologist affiliated to the Neuromuscular Reference Centre for Children at the University Hospitals Leuven, Belgium. She devoted her carrier to the multidisciplinary management of patients with neuromuscular disorders and to research in therapy development for Duchenne muscular dystrophy and spinal muscular atrophy.

Yoram Nevo
Debate Speaker

Schneider Children's
Medical Center of Israel

Prof. Yoram Nevo is a paediatric neurologist and currently the director of the Institute of Neurology of Schneider Children's Medical Centre of Israel. He was trained in neuromuscular disorders at Washington University, St. Louis and, since then, has been involved in Israel in clinical practice, translational and clinical research in this field. He is the head of the Israel Paediatric Neurology Association.

Kathryn Swoboda
Debate Speaker

Massachusetts General
Hospital, USA

Kathryn J. Swoboda is the Katherine B. Sims M.D. Endowed Chair in Neurogenetics and Director of the Neurogenetics Unit, Center for Genomic Medicine, Massachusetts General Hospital

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