MDA has invested over $125M in the development of gene therapy for neuromuscular diseases (NMDs) over the past 20 years. To continue to lead the way in this important field of medicine, MDA held a summit in January 2022 to address the challenges in safely delivering AAV-based gene replacement therapy to NMD patients. In this meeting, top experts and stakeholders convened to talk openly about adverse events linked to clinical trials in the field. Although experts collectively agree that gene therapy holds great promise in addressing the underlying cause of many neuromuscular diseases, there are still many challenges and risks that must be overcome to fully realize the translational potential of gene therapy in NMDs affecting children and adults.