Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities

Dr Goyenvalle is a director of research at INSERM and head of the laboratory Biotherapies for neuromuscular diseases at the University of Versailles. Her research projects focus on gene and antisense therapies for the treatment of neuromuscular disorders. She notably demonstrated the therapeutic potential of novel antisense tools for the treatment of Duchenne muscular dystrophy, that may offer the exciting opportunity to treat both the dystrophic muscle phenotype and the brain comorbidities in DMD patients. Dr Goyenvalle obtained her PhD in Virology at the University of Paris VII in 2006 and then moved to the University of Oxford as a post-doctoral scientist to investigate various splicing modulation approaches for neuromuscular and neurodegenerative diseases. In 2012, she was awarded a Chair of Excellence program to establish her own group at the University of Versailles to develop novel RNA based technology for the treatment of neuromuscular diseases.