Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy.
[3] It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells.
[1] The most commonly reported side effects include vomiting, nausea, acute liver injury, pyrexia (fever), and thrombocytopenia (abnormally low platelet count in the blood).
FDA states that the conditional approval is based on detection of successful gene expression; evidence of clinical improvement is still pending.
[3] In the phase 3 randomized clinical trial published in Nature Medicine, Elevidys AAV gene therapy for Duchenne muscular dystrophy failed to show statistically significant motor function improvement.