It is an incurable genetic disease, no treatment exists to cure this pathology. But a first child has received a candidate treatment in France, this clinical trial is a great first.
Myopathy is a group of pathologies that mainly affect the muscles. Myopathy is likely to be genetic. The forms of the disease can be variable. The best known being Duchenne muscular dystrophy, characterized by progressive degeneration of muscle cells … in patients, smooth muscles and heart muscle are damaged with each contraction … with wear and tear, they end up destroying themselves.
This rare pathology affects around 2,500 French people, 99% of whom are male, which represents 1 boy in 3,500. But the situation could change with the help of a young boy (whose age has not been communicated). ) with Duchenne muscular dystrophy. He was treated with gene therapy at I-Motion, in Paris. It is the first of an international multicenter phase I / II / III trial, of which Généthon, the AFM-Telethon laboratory, is the promoter. “We are sorely lacking in treatments for Duchenne muscular dystrophy to date. We have high hopes for this drug candidate.”, confides Prof. Muntoni, of the Dubowitz Neuromuscular Center (London, United Kingdom) through a Press release published by the Généthon laboratory.
Duchenne muscular dystrophy is the most common neuromuscular disease in children
The research laboratory created thirty years ago with funding from the Telethon, provides details on this gene therapy. The principle of the latter is based on an intravenous injection which would provide “a shortened version of dystrophin, the protein deficient in people with Duchenne muscular dystrophy”.
Other gene therapy trials on Duchenne muscular dystrophy are currently being undertaken worldwide. “ Duchenne muscular dystrophy is a particularly complex disease because it affects all muscles. We must therefore remain cautious but we are proud that the technologies developed at Généthon are now becoming drug candidates likely to change the future of patients “, says Frédéric Revah, General Manager of Généthon.
