Francesco Muntoni is a Professor of Paediatric Neurology and the Director of the Dubowitz Neuromuscular Centre, at the UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK. In the Institute and hospital he led the Novel Therapies Programme of the Biomedical Research Centre and between 2008 and 2022 and also the Developmental Neuroscience Programme between 2008 and 2018.
Since 2022 Muntoni is also director of the Genetic Therapy Accelerator Centre, a new gene therapy translational research partnership , based at Queen Square Institute of Neurology and in close collaboration with the UCL Great Ormond Street Institute of Child Health. This cross-faculty collaboration within UCL will drive forward an exciting evolving area with direct therapeutic benefits for patients with disabling neurological conditions.
Muntoni has an interest in pathogenesis, deep phenotyping, gene identification for rare neuromuscular conditions and translational research in Duchenne muscular dystrophy, spinal muscular atrophy and congenital myopathies. He is involved in several natural history studies and clinical trials. His research funded by the Department of Health, MRC and the European commission lead to the development and early clinical trials of 2 morpholino antisense
oligonucleotides, now approved by FDA, that induce partial correction of the processing defect of the DMD gene in boys with Duchenne muscular dystrophy. He is the UK chief investigator for multiple clinical trials on DMD and SMA, including genetic therapies involving antisense oligonucleotides and AAV delivered transgenes.
In the last few years he obtained several international awards, including the 2022 Ottorino Rossi Award, the World Duchenne Organisation 2022 Leadership Awards, the 2023 European Paediatric Neurology Society Jean Aicardi Award and the 2024 Muscular Dystrophy UK President Award.
Web sites:
https://www.ucl.ac.uk/child-health/research/developmental-neurosciences/molecular-neurosciences/dubowitz-neuromuscular-centre
https://www.northstardmd.com/
http://www.smareachuk.org/
www.bindproject.eu
https://www.ucl.ac.uk/child-health/news/2022/sep/new-gene-therapy-translational-research-partnership-between-institutes-child-health
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