A new milestone has been reached in the clinical trial of gene therapy conducted by the Généthon laboratory for Duchenne muscular dystrophy.
Building on the positive results of the dose escalation phase, which demonstrated the safety and efficacy of the second dose tested, Genethon has just obtained authorization from the European Medicines Agency (EMA) (with France (ANSM) as the reporting member state) and the United Kingdom (MHRA) to start the pivotal phase of the trial.
The first patients will be enrolled in August and September in the United Kingdom and France for this pivotal phase, conducted as a double-blind trial, which will involve 64 boys aged 6 to 10 with Duchenne muscular dystrophy who have retained their ability to walk.
