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Lunch Hour Lecture | Developing therapies for muscular dystrophy and spinal muscular atrophy
25 October 2022, 1:00 pm–2:00 pm
In this Lunch Hour Lecture Professor Francesco Muntoni will discuss Developing therapies for muscular dystrophy and spinal muscular atrophy.
This event is free.
Event Information
Open to
- All
Availability
- Yes
Cost
- Free
Organiser
-
UCL Events
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About the lecture:
The lecture will focus on the way the expression of genes with pathogenic variants can be manipulated to generate the desired outcome in children with the most common and severe neuromuscular conditions, Duchenne muscular dystrophy and Spinal muscular atrophy. Professor Muntoni will also briefly discuss the recent developments in the field of adeno associated viral gene therapy for spinal muscular atrophy.
This Autumn, we bring you a Lunch Hour Lecture Series to showcase how UCL research transforms lives.
About the Speaker
Professor Francesco Muntoni
Professor of Paediatric Neurologist and the Director of the Dubowitz Neuromuscular Centre at UCL
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 is Theme Lead in the Novel Therapies of the Biomedical Research Centre and between 2008 and 2017 led the Developmental Neuroscience Programme. Since 2008 he is the co-directed 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 DMD, SMA 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 clinical trials on DMD and SMA, including AAV trials.