Mothers of Duchenne muscular dystrophy patients provide poignant testimony amid escalating fatalities linked to gene therapies. They underscore the harsh realities faced by families confronting a fatal diagnosis and the difficult decisions surrounding experimental treatments fraught with risks such as liver failure. Advocating for continued clinical advancements, these caregivers highlight that despite setbacks, approved therapies and robust pipelines offer renewed hope for altering Duchenne’s bleak prognosis.