Meeting report: 2025 muscular dystrophy association summit on ‘safety and challenges in gene therapy of neuromuscular diseases’

The 2025 MDA Gene Therapy Summit brought together leading researchers, clinicians, and industry representatives to discuss the latest advancements and challenges in the development and clinical translation of genetic therapies for neuromuscular disorders. The meeting featured in-depth presentations on clinical trials, therapeutic developments, and regulatory pathways for AAV-mediated gene therapies, highlighting both successes and challenges in the field.