Duchenne muscular dystrophy (DMD), the most common form of myopathy in children, is currently experiencing major developments in innovative therapies. This neuromuscular disease leading to loss of walking in early adolescence and early death in adulthood, can thus benefit, still experimentally in most cases, from different therapeutic approaches such as exon(s) skipping (by antisense oligonucleotides) or gene transfer (by injection of a transgene encoding a mini- or microdystrophin and conveyed by a viral vector). The latter technique is the subject of real risk-taking, including at the vital level, as demonstrated by past or current clinical trials in other pathologies.
It is in this context that a study, published in March 2021, on the initiative of American patient associations, tried to measure the degree of acceptability of this risk-taking. The questionnaire returned by 285 caregivers and 35 patients with DMD assumed that gene therapy could only slow the progression of the disease and that it would last for up to ten years. The question was also about the different times in life when it might apply. The results show that the more people are advanced in their disease, the more they are inclined to accept risk taking. The same response profile is observed both in patients and their caregivers.
