The FDA’s decision to ask Sarepta to stop shipping its gene therapy Elevidys for Duchenne muscular dystrophy is a sobering reminder of the risks and uncertainties involved in treating rare and fatal diseases. The therapy, which was hailed as a breakthrough just months ago, has now been linked to two deaths in non-ambulatory patients.
For families like mine, who have lost loved ones to this devastating disease, every single day is a harsh reality check. We’ve watched our children struggle with muscle weakness and fatigue, their bodies slowly failing them. The prospect of losing them to the disease before their time is unbearable.
But we’re also grateful for the progress that’s been made in recent years. Clinical trials, FDA-approved therapies, and a pipeline rich with additional prospects have given us hope and control over our children’s treatment options. It’s a tiny asterisk on the typical Duchenne trajectory chart, but it’s enough to make all the difference.
As parents, we’ve had to make tough decisions about our children’s care, weighing the risks and benefits of experimental treatments like Elevidys. We’ve signed lengthy consent forms acknowledging the potential side effects, including liver injury and death. But we’ve also had to decide whether to wait for more information or act now, knowing that every day counts.
The recent deaths have shaken us, but they’re not a reason to roll back progress. Instead, they’re an opportunity to learn from our mistakes and improve the therapies that are saving lives. The FDA’s decision is a reminder that risk management is crucial in developing new treatments for rare diseases.
As we move forward, it’s essential that families, clinicians, policymakers, and industry work together to apply lessons learned from new therapies – both successes and setbacks – to sustain and multiply progress. We mustn’t forfeit our privilege of risk to knee-jerk wishes to remove risk entirely. Instead, we need to harness the power of choice and access for patients like ours who lack the luxury of time.
In rare disease, risk is a privilege that we must continue to exercise. It’s a reminder that every decision we make – whether it’s about treatment options or research priorities – has the potential to save lives. Let’s work together to ensure that our children have access to the best possible care, even if it means navigating uncertain risks and outcomes.
Source: https://www.statnews.com/2025/07/18/sarepta-elevidys-deaths-duchenne-muscular-dystrophy-dmd-mothers-research-progress