Developing therapies for muscular dystrophy and spinal muscular atrophy

Date of Lecture: Tuesday 25 October 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. About the speaker: Professor Francesco Muntoni , Professor of Paediatric Neurologist and the Director of the Dubowitz Neuromuscular Centre at UCL