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 the 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 is driving forward an exciting evolving RNA and AAV gene therapy 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.

Muntoni is the chief investigator of the European consortium BIND (Brain Involvement in Dystrophinopathies, https://bindproject.eu/) focusing on the brain comorbidities observed in a proportion of patients with Duchenne muscular dystrophy, and exploring both their biological basis, clinical assessment tools and preclinical translational research.

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, the 2024 Muscular Dystrophy UK President Award, and the 2025 Novo Nordisk 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