As treatment with delandistrogene moxeparvovec begins to be widely prescribed in the United States for Duchenne muscular dystrophy (DMD), American experts have come together to issue recommendations aimed at better identifying and preventing the deleterious effects of this gene therapy (GT) on the heart:
- cases of myocarditis induced by TG remain exceptional but can be fatal if they go undetected,
- cardiac monitoring (clinical, biological and imaging) is therefore essential,
- this consensus work was carried out with cardiologists on the basis of their real-life experience,
- the experts also emphasise the risks incurred by children receiving this TG who already have a heart condition,
Ultimately, these very detailed recommendations argue in favour of a relative restriction on the indications for TG in this at-risk population, and the need for a degree of caution.
