Seminar: Developing therapies for muscle and brain in Duchenne muscular dystrophy
Prof Francesco Muntoni, Professor of Paediatric Neurologist and Director of the Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children
Thursday, 09 November 2023, 4pm to 5pm
Institute of Developmental and Regenerative Medicine (IDRM) IMS-Tetsuya Nakamura Building Old Road Campus, Roosevelt Drive, Oxford, OX3 7TY
Hosted by Professor Matthew Wood
MDUK OXFORD NEUROMUSCULAR CENTRE SEMINAR SERIES
This seminar is intended for the personnel and students at the University of Oxford and the Oxford University Hospitals Foundation Trust.
Hybrid seminar at the IDRM - IMS-Tetsuya Nakamura Building on Old Road Campus. For the online link, please email firstname.lastname@example.org.
All attendees are invited for a drinks reception immediately following the talk.
A number of different therapeutic strategies are under development to improve outcome in DMD, some having received conditional approval in some countries, others at different stages of development.
In this lecture I will provide an overview of the status of some of the most promising therapies under development to improve muscle function in DMD. I will also present the work that our consortium (BIND Consortium, www.bindproject.eu) is doing to consider the implication of genetic therapies to also address the deficiency of the defective gene in brain.
Francesco Muntoni is a Professor of Paediatric Neurologist 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 led the Developmental Neuroscience Programme between 2008 and 2018.
Since 2008 he is the co-director of the MRC Centre for Neuromuscular Diseases at UCL.
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 2022 Muntoni was made 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.
In the last few years he obtained several international awards, including the 2022 Ottorino Rossi Award, the World Duchenne Organisation 2022 Leadership Awards and the 2023 European Paediatric Neurology Society Jean Aicardi Award.