Sirolimus for the treatment of steroid-refractory hepatotoxicity following AAV gene therapy in patients with Duchenne muscular dystrophy
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Background: Adeno-associated virus (AAV)-mediated gene therapy with delandistrogene moxeparvovec-rokl (Elevidys®) is an approved treatment for patients with Duchenne muscular dystrophy (DMD). While generally well tolerated, hepa... ...
