McGill University, Montreal , Canada

Dr. Maryam Oskoui is an epidemiologist and pediatric neurologist. She is Director of the Pediatric Neurology Division at the Montreal Children’s Hospital and a Senior Clinical Research Scholar of the FRQ-S with a focus in pediatric neuromuscular disorders. She is the Principal investigator and SMA lead for INFORM RARE, a CIHR Strategy for Patient Oriented Research innovative clinical trial network. Dr. Oskoui serves as an evidence-based methodologist for the American Academy of Neurology, contributing to clinical care guidelines.

University of Arkansas for Medical Sciences, Little Rock, AR , United States

Aravindhan Veerapandiyan, MD is a Child Neurologist and Assistant Professor with specialized interest and training in neuromuscular disorders in children. He is the Director of the Comprehensive Neuromuscular Program and Co-Director of the Muscular Dystrophy Association Care Center at Arkansas Children’s Hospital / University of Arkansas for Medical Sciences. Dr. Veerapandiyan earned his medical degree from the K.A.P. Vishwanatham Government Medical College in India. He completed a residency in Child Neurology at Rutgers University – New Jersey Medical School in Newark, New Jersey and a fellowship in Neuromuscular Medicine at University of Rochester – Strong Memorial Hospital in Rochester, New York.

Leiden University Medical Center, Leiden , Netherlands

Annemieke Aartsma-Rus is a professor of translational genetics at the Leiden University Medical Center. She has been involved in setting up guidelines for the use of Duchenne muscular dystrophy mouse models in association with TREAT-NMD.

Sorbonne Université, GHU Pitié-Salpêtrière Hospital, AP-HP, Paris, France, Paris , France

Dr Teresinha Evangelista is a Board-certified neurologist and neuropathologist with long standing clinical and research expertise in neuromuscular disorders. Dr Teresinha Evangelista was the president of the Portuguese Society for the Study of Neuromuscular Diseases from 2009 to 2013. In 2018, she became the coordinator of the European Reference Network for rare neuromuscular diseases EURO-NMD. In 2018, she joined the « Unité de Morphologie Neuromusculaire; Institut de Myologie - Sorbonne Université, GHU Pitié-Salpêtrière ».

University of Florida, Gainesville , United States

With over 10 years of research experience in molecular biology, neuroscience, rare disease treatment, and AAV gene therapy, I am an Assistant Scientist at the University of Florida, where I investigate and promote basic science translation into effective therapies for genetic disease. I have a Ph.D. in Biology from the University of Vermont, multiple certifications in biopharmaceutical manufacturing and clinical trials, and one patent on materials and methods for treatment of Friedreich's Ataxia. My core competencies include developing and running robust cell-based assays using high-throughput detection technologies such as fluorescence, mitochondrial assays, and flow cytometry. I have managed and contributed to a broad portfolio of target-focused projects in academia and industry centered around rare diseases such as spinal muscular atrophy, EMARDD, and Barth disease. I have also developed partnerships and collaborations across the department, influencing project strategies and platform development. My overarching research goal is to advance gene therapy-based strategies to control motor deficits and enhance neuromuscular regeneration.

Charité – Universitätsmedizin Berlin, Berlin , Germany

Werner Stenzel is a Neuropathologist and Myopathologist, and the head of the neuromuscular diagnostic and research unit in Berlin at the Charité Hospital.