Francesco Muntoni, M.D., is a Professor of Paediatric Neurology who trained in Italy but has worked in the UK since 1993. He is the Director of the Dubowitz Neuromuscular Centre, at the Great Ormond Street UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK. He led the Developmental Neuroscience Programme between 2008 and 2018, and was also Theme Lead in the Novel Therapies of the Biomedical Research Centre in the Institute and Great Ormond Street Hospital between 2008 and 2022. Since 2022 he has served as the Director of the Genetic Therapy Accelerator Centre, in the Department of Neurodegenerative Diseases, in the UCL Queen Square Institute of Neurology. This cross-faculty collaboration is focused on novel genetic therapies for neurological disorders. Dr. Muntoni’s main interests are in pathogenesis, deep phenotyping, novel genes identification and translational research aspects in pediatric neuromuscular disorders, especially Duchenne muscular dystrophy and spinal muscular atrophy. He is currently involved and leads several natural history studies and clinical trials using antisense oligonucleotide approaches and AAV gene therapy. His efforts in the last 20 years led to two FDA-approved therapies for Duchenne muscular dystrophy (eteplirsen and golodirsen). Regarding gene identification, in collaboration with colleagues from UK, Europe, USA, and Australia, he has been involved in the identification of more than 30 neuromuscular disease genes.