American specialists in gene therapy using adeno-associated viruses (AAV) have investigated the serious cases of liver toxicity that occurred during gene therapy with microdystrophin (delandistrogene moxeparvovec, Elevidys®) in patients with Duchenne muscular dystrophy:
- four patients treated with this product who developed serious liver damage on average one month after injection were given higher doses of corticosteroids as well as oral rapamycin (sirolimus);
- thanks to this enhanced immunosuppression protocol, the patients, one of whom was already in a wheelchair, regained normal liver function after two months.
This molecule, which is already used for other indications, could soon be included in the recommendations for this condition.
