Speakers

Opening Lecture

COVID-19 for NMD, telemedicine, changing practice in the pandemic situation.

The COVID-19 pandemic has so far not been the largest, or the most lethal pandemic of the last 100 years, but the global response to it has been unprecedented, both in medical and in societal terms. It has led to a significant change in behaviour, specifically in the way medicine is practised, and its longer- term effects cannot yet be predicted confidently. The concerns for people with neuromuscular disease specifically are significant: in addition to potentially being a risk group for severe infection, they are put at risk by an interruption to their access to treatment. The World Muscle Society is making an effort to help them face these challenges, by providing advice for patients and carers, trying to define best practice parameters, and suggest avenues to manage the future changes in medical practice. This lecture discusses the approach, its effects on patients, and potential future developments in telemedicine.

Dr Maxwell Damian
Speaker

Maxwell S. Damian, MD, PhD (Habil), FNCS, FEAN Cambridge University Hospitals.
Max is an Australian neurologist and works in Germany and Cambridge, UK. His clinical and academic work has centred on neurointensive care and on the acute and emergency management of neuromuscular disease. He has been a member of the WMS since 2004 and is a Fellow of the Neurocritical Care Society and the European Academy of Neurology, where he chaired the Neurocritical Care Scientific Panel 2015-2020. He was the principal author of the WMS advice and practice parameter papers.

Jiri Vajsar
Session Moderator

Jiri Vajsar MD, MSc, FRCPS.The Hospital for Sick Children (SickKids) and The University of Toronto, Canada
Jiri has a long-standing interest and expertise in paediatric neuromuscular diseases and he has been a member of the WMS for 25 years. His research and publications deal with SMA, muscular dystrophies, GBS and MG and his research has been funded by MDAC, NIH, SMA Foundation and Industry.

Panel discussion 1

Pre-clinical requirements for novel neuromuscular therapies

It is a unique time for neuromuscular disorders, as an unprecedented number of therapeutic candidates are entering clinical trial for a range of diseases. With this surge of potential treatments, the importance of pre-clinical models and rigor in pre-clinical drug development has never been more important. It is critical to evaluate study designs, predictive power, reliability and relevance of current animal models in assessing the efficacy and safety of emerging therapies. In this panel, we have assembled four world leading experts on the application of pre-clinical models to therapy development. We will present key ideas related to best practice and discuss important pitfalls that have plagued pre-clinical drug programs. We will also identify current gaps in the field and offer ideas for future improvements.

Jim Dowling
Session Moderator

Dr James Dowling, Clinician-Scientist, The Hospital for Sick Children (SickKids); University of Toronto, Canada.
Dr Dowling received his BSc from Yale, his MD/PhD from University of Chicago, and completed residency at Children’s Hospital of Philadelphia. Dr. Dowling’s clinical expertise is in neuromuscular disorders, and his research examines disease pathogenesis and therapy development for congenital myopathies.

Grace Yoon
Content Moderator

Grace Yoon, Staff physician in the Division of Clinical and Metabolic Genetics, The Hospital for Sick Children (SickKids); University of Toronto, Canada
She and Dr. Jim Dowling co-direct a multidisciplinary clinic which is dedicated to the care of children with genetic neuromuscular disorders.

Professor Annamaria DeLuca
Panel Speaker
University Of Bari, Dept Pharmacy - Drug Sciences

Pharmacologist at the University of Bari. Main research interest: pre-clinical studies for identification and validation of druggable targets and personalized medicine in models of inherited and acquired neuromuscular conditions, such as muscular dystrophies, channelopaties, sarcopenia. Member of Executive Committee of TREAT-NMD and Chair of TREAT-NMD Advisory Committee for Therapeutics (TACT).

Professor Jennifer Morgan
Panel Speaker
Ucl Great Ormond Street Institute Of Child Health

Jennifer Morgan is Professor of Cell Biology in the Dubowitz Neuromuscular Centre at University College London Great Ormond Street Institute of Child Health. Her main research interests are skeletal muscle stem cells and skeletal muscle regeneration.

Professor Kanneboyina Nagaraju
Panel Speaker
Binghamton University

Dr. Kanneboyina Nagaraju is a tenured Professor and founding Chair at the SUNY-Binghamton University. He is an expert on translational research on autoimmune and genetic muscle diseases. He co-founded two biotechnology companies, ReveraGen BioPharma Inc, and Agada Biosciences. Dr. Nagaraju has authored over 140-publications and has mentored over 80 fellows.

Professor Dominic Wells
Panel Speaker
Royal Veterinary College

Professor Dominic (Nic) Wells is a qualified veterinary surgeon with a PhD in physiology. He currently works at the Royal Veterinary College, London where he is Professor in Translational Medicine. Major research interests are animal welfare and testing potential therapies for neuromuscular diseases in preclinical animal models.

Panel discussion 2

Gene/ advanced/ new therapeutics

More information to follow

Kathryn Wagner
Session Moderator

Kathryn R. Wagner, M.D., Ph.D. Professor of Neurology and Neuroscience at the Johns Hopkins School of Medicine and Director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute.
She is advisor for Parent Project Muscular Dystrophy, the FSHD Society, the TREAT-NMD Advisory Committee on Therapeutics and an elected member of the American Society for Clinical Investigation.

Chris Weihl
Content Moderator

Dr. Weihl, Professor of Neurology at Washington University School of Medicine in Saint Louis, Missouri.
His research program focuses on both genetic and acquired myopathies with an emphasis on how dysfunction in protein quality controls lead to muscle degeneration. Clinically, Dr. Weihl sees patients in a multidisciplinary muscular dystrophy association clinic and recently established a clinic devoted to the care of patients with sIBM.

Speakers

Dr Ana Buj Bello
Panel Speaker
Genethon

Ana Buj Bello is research director at INSERM and leads a team at Genethon, France. She received a degree in Medicine and Surgery and a PhD in Neurosciences. Her research activities focus on developing AAV-based gene therapies for neuromuscular disorders, with a major interest in clinical translation.

Professor Carsten Bönnemann
Panel Speaker
National Institutes of Health, USA

Carsten Bönnemann graduated from Medical School in Freiburg/Germany. He trained in child neurology, neuromuscular and genetics at MGH and Boston Children’s Hospital. He is on faculty at Children's Hospital of Philadelphia and in 2010 was recruited to the NINDS/NIH as Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section.

Dr Barry Byrne
Panel Speaker
University Of Florida

Dr. Byrne is a clinician scientist studying a variety of rare diseases with the specific goal of developing therapies for inherited cardiac and skeletal muscle disease. The research team has developed new therapies using AAV-mediated gene therapy in DMD, Friedreich’s ataxia, Pompe and other inherited neuromuscular diseases. Dr. Byrne is the Associate Chair of Pediatrics, Director of the Child Health Research Institute and the Earl and Christy Powell University Chair in Genetics.

Professor Francesco Muntoni
Panel Speaker
University College London

Professor Muntoni is a paediatric neurologist with an interest in deep phenotyping and genetic basis of paediatric neuromuscular disorders, and with expertise in natural history and translational research aspects of several conditions affecting children.