Dr. Villalta is an Associate Professor in Physiology and Biophysics and Founding Director of the Muscle Biology and Disease Research Center at the University of California Irvine. His research programme aims to define how the immune system regulates the pathogenesis of inherited and acquired myopathies.

Dr. Tahseen Mozaffar is a Professor of Neurology and Pathology and Laboratory Medicine and the Director of the Division of Neuromuscular Disorders. Dr. Mozaffar serves as chair of one of the biomedical committees  and is the Associate Director for the Center for Translational Sciences Award (CTSA) at University of California, Irvine. He is the Principal Investigator for UCI-NEXT, the NeuroNEXT award to the University of California, Irvine, one of 25 such NeuroNEXT sites funded by the NINDS/NIH. He is also the Lead Investigator for a multicenter NIH/NIAMS funded Natural History Study in sIBM (INSPIRE-IBM), which started April 2021.

He graduated medical school at the Aga Khan University in Karachi, Pakistan in 1989 and trained in Neurology and Neuromuscular Disorders at Washington University/Barnes Hospital in St. Louis, MO. Dr. Mozaffar has been at UC Irvine since 2000 where he has built an internationally recognized clinical and research program in Neuromuscular Disorders.

He is actively involved in clinical and translational research in Neuromuscular Disorders, including currently serving as Principal Site Investigator on over a dozen clinical trials in myasthenia gravis, rare and ultra-rare myopathies and in immune myopathies. He has co-authored over 190 peer-reviewed publications and has authored or co-authored over a dozen book chapters and invited reviews. As an expert in these rare and ultra-rare myopathies, he is actively sought as an advisor by pharmaceutical companies for trial design and identifying disease targets.

He is the Director of the nationally recognized Annual UC Irvine Neuromuscular Colloquium, now in its 12th year of existence and the founding Director of the Annual Neuromuscular Pathology Colloquium, now in its 7th year.

Jiří Vencovský is a professor of internal medicine at the Institute of Rheumatology and the Department of Rheumatology at the 1st Medical Faculty of Charles University in Prague.

He is the head of the research department at the Institute. He is a former long-time chairman of the Czech Society of Rheumatology and editor-in-chief of the journal Czech Rheumatology. For six years he served as Treasurer of the Foundation for Research in Rheumatology (FOREUM) and is currently a member of the FOREUM Board of Trustees.

His research interests include idiopathic inflammatory myopathies, autoantibodies in rheumatic diseases and treatment of rheumatoid arthritis. He is one of the founding members of the European Registry of Inflammatory Myopathies and has hosted a number of meetings on this topic, most recently the 4th Global Conference on Myositis (GCOM) in 2022.

Dr Sithara Ramdas is a Consultant Paediatric Neurologist based at the Oxford Children’s Hospital and Honorary senior lecturer, University of Oxford. She leads the Paediatric Neuromuscular Service and is the paediatric lead for the National Congenital Myasthenia Service. Her specialist clinical and research focus are on neuromuscular disorders and neuroimmunology.

Olga Germanenko is patient advocate from Russia. She is the founder and current President of Russian SMA Family Foundation and Vice-President of SMA Europe. Her relation to neuromuscular disorders is personal. She is the mother of a 15 year old daughter living with spinal muscular atrophy type1.

SMA Europe is a non-profit umbrella organisation of spinal muscular atrophy (SMA) unites 29 patient organizations from 27 countries. SMA Europe targets to bring effective treatments and optimal care to everyone living with SMA.

SMA Family Foundation Russia is a national non-profit patient organization unites more than 1,450 families living with SMA across the country. SMAFF provides patient support services, educational activities, advocacy initiatives, working hard for improving the care in SMA with all relevant stakeholders.

Olga also serves as a TREAT-NMD Education Committee member, and is a member of numerous other advisory and expert boards (government, industry, societal).

Josie Godfrey has worked in rare diseases and innovative therapies for over 15 years. She currently runs a consultancy business specialising in strategic market access, policy and stakeholder engagement. She is the Strategic Director for Duchenne UK’s Project HERCULES, an award-winning global collaboration developing evidence and tools to support HTAs for new treatments for Duchenne Muscular Dystrophy. Josie is also co-founder and joint CEO of Realise Advocacy, which supports patient involvement in drug development and access processes. She previously led work at NICE to establish the Highly Specialised Technologies programme.

Pedro M Rodriguez Cruz is a neurologist with special interest in neuromuscular and neurogenetic diseases in underrepresented populations of Sub-Saharan Africa. He focuses on diagnostics, genetic variation, phenotypic variability and gene discovery.

Dr. Soledad Monges did her paediatric neurology residency and a fellowship training in neuromuscular disorders at Hospital Garrahan, where she established a Clinic for Neuromuscular Diseases and is currently Chair of the Department of Paediatric Neurology. She participated in clinical trials for the diagnosis of neuromuscular diseases.

Venugopalan Y Vishnu is an academic neurologist working as Additional Professor at the  All India Institute of Medical Sciences, New Delhi, India.

Jeannine Heckmann is a professor of neurology at the University of Cape Town, South Africa. Her research interests include neuromuscular conditions in the African context and include myasthenia gravis, motor neuron diseases, and inherited neuromuscular conditions.

Professor Swanson is interested in RNA regulatory pathways during mammalian development and how this regulation is disrupted in neurological and neuromuscular diseases, including some types of muscular dystrophy and amyotrophic lateral sclerosis. He received his PhD from the University of California, Berkeley, and is currently a Professor in the Department of Molecular Genetics and Microbiology at the University of Florida College of Medicine and Associate Director of the Center for NeuroGenetics. His lab focuses on the functions of repetitive DNA elements, particularly microsatellites or short tandem repeats (STRs), in RNA-mediated disorders. An important objective of these studies is to enhance tissue regeneration following treatment modalities designed to block the toxicity of STR RNAs.

Grace Yoon is currently a staff physician in the Division of Clinical and Metabolic Genetics, with a cross appointment in the Division of Neurology at the Hospital for Sick Children in Toronto, Canada. In addition to providing clinical care for patients with inherited neurological disorders, she is an active teacher within the Faculty of Medicine at the University of Toronto. She developed a neurogenetics elective rotation which provides trainees from the divisions of Neurology and Clinical and Metabolic Genetics with unique educational opportunities, including participation in a multidisciplinary clinic between the divisions of Paediatric Neurology and Clinical Genetics, which is dedicated to the care of children with genetic neuromuscular disorders. The main goal of her research is to define the genetic basis of complex inherited neurological disorders, with genetic neuromuscular disorders and inherited ataxias being areas of particular focus.

Pietro Spitali is an associate professor at the Human Genetics department of the Leiden University Medical Center in the Netherlands. His lab focuses on identification of biomarkers in neuromuscular disorders in pre-clinical and clinical studies. He is also actively researching the basic mechanism of gene expression regulation in muscle by studying RNA binding proteins, pre-mRNA synthesis and gene expression at spatial resolution. He is currently co-coordinator of Fluid Biomarker workgroup of the Duchenne Regulatory Science Consortium (D-RSC) of the Critical Path Institute, member of the TREAT-NMD Neuromuscular Disease Advisory Committee (NMDAC) and founding member of the Netherlands single-cell network. https://nmd-biomarkers.org/

Dr Arechavala-Gomeza leads the Nucleic Acid Therapeutics for Rare Diseases group at Biobizkaia HRI. She participated in the development of eteplirsen for DMD and she has led a network of stakeholders in the development of better delivery options for these therapies for target tissues.