Given the rapid and recent development of microdystrophin gene therapy treatments for Duchenne muscular dystrophy (DMD), there is little data on the side effects of these treatments.
- The Delphi method was used to develop a consensus on the assessment and management of side effects (vomiting, gastritis, acute liver damage, myocarditis, autoimmune myositis) of delandistrogene moxeparvovecavant (Elevidys®).
- The experts consulted all had experience of administering this product as part of clinical trials or in the management of boys with DMD or gene therapy side effects.
- This consensus of experts constitutes a basis for work and exchanges to improve the management of potential adverse effects of microdystrophin gene therapy.
