This year’s World Duchenne Awareness Day theme is ‘Family: At the Heart of Care’. A documentary is available on this theme, following families from around the world who are living with Duchenne muscular dystrophy.
Research into Duchenne muscular dystrophy is gathering pace
This day is an opportunity to highlight the accelerated development of innovative therapies for this neuromuscular disease. Gene therapy represents a very promising avenue for the treatment of this rare genetic disease. At the end of July, following positive results in phases I/II/III, Généthon obtained the necessary authorisations to begin the pivotal phase of the trial. This double-blind phase will involve a total of 64 boys aged 6 to 10 with Duchenne muscular dystrophy who have retained their ability to walk. The clinical trial centre, I-Motion, at the Institute of Myology will be one of the reference centres.
Duchenne muscular dystrophy research at the Institute
Within the Center of Research in Myology, France Pietri-Rouxel heads the Muscle mass and function maintenance & optimization of AAV-based gene therapies Group (MOOVE) group, which aims to optimise the therapeutic approach for treating Duchenne muscular dystrophy, to characterise the phenotypic and genomic characteristics of Becker patients carrying the deletion of exons 45 to 55, and to decipher the mechanisms governing skeletal muscle plasticity, an essential step in understanding its physiopathological processes.
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