Elevidys gene therapy has shown promising results in clinical trials for DMD. However, questions remain about its real-world effectiveness and its approximately $3 million price tag, which is widely considered unaffordable for most families. Many are forced to organize individual fundraising campaigns to access treatment.
In this interview, we explore key concerns, unanswered questions, and broader implications for global access to innovative therapies. Read More: FAQs About Elevidys
How much can Elevidys extend a child’s life expectancy?
This question remains difficult to answer definitively. Elevidys was approved by the FDA in the summer of 2024, meaning that long-term data on life expectancy is still limited.
The true impact of the treatment on survival can only be understood over time as more patients are monitored in the coming years. At present, no one can say with certainty how much Elevidys may extend a child’s lifespan.
However, certain biomarkers — such as CK (creatine kinase) levels and dystrophin expression levels — may provide some indication of whether the therapy is helping slow disease progression. Even so, the long-term clinical significance of these findings has not yet been fully established.
Is Elevidys, priced at $3 million, truly worth the cost?
No. In my opinion, the price is far too high. A treatment that costs $3 million should demonstrate clear, measurable, and long-term clinical benefits. However, there are still major unanswered questions surrounding Elevidys, including how much it can extend life expectancy, how significantly it slows disease progression, and whether the improvements seen in clinical trials are truly meaningful in everyday life.
Many families around the world are being pushed into desperate fundraising campaigns based on hope rather than definitive evidence. While gene therapy is an important scientific advancement, the current price of Elevidys creates serious ethical concerns about accessibility, transparency, and the commercialization of rare diseases. In my view, no treatment should carry such an enormous financial burden unless its long-term effectiveness is clearly proven. Read More: Why Duchenne Muscular Dystrophy Treatment Costs Are Out of Control
Why do you think Elevidys gene therapy is so expensive?
In my opinion, one of the biggest problems is the lack of transparency between pricing and real clinical benefit. Sarepta Therapeutics presents Elevidys as a groundbreaking therapy, yet families are still unable to receive clear answers about its long-term impact on survival, quality of life, or disease progression.
As a father of a child with Duchenne muscular dystrophy, I believe parents deserve more than hope and marketing language. A treatment priced at $3 million should come with strong, transparent, and indisputable evidence showing how much it can truly help patients over the long term. Instead, many families are left trying to raise enormous amounts of money while still facing uncertainty about the actual benefits of the therapy.
I also believe the pricing reflects a growing commercialization of rare diseases, where desperate families can become trapped between hope and financial pressure. Scientific innovation is important, but no therapy should be promoted at such an extraordinary cost without fully answering the most important question for families: What meaningful difference will this make in a child’s life?
Do you have concerns about the clinical effectiveness of Elevidys?
Yes, I do have serious concerns about the clinical effectiveness of Elevidys. Supporters of the therapy often focus on NSAA scores, but for many families, the most important issue is whether the treatment creates meaningful and measurable improvements in the real progression of the disease. Biomarkers such as CK, AST, and ALT levels are extremely important because they can provide insight into ongoing muscle damage and overall disease activity.
If a therapy costs $3 million, families deserve transparent and detailed data showing how significantly these biomarkers improve and whether those changes are sustained over time. At the moment, there is still uncertainty about how biomarker improvements translate into long-term functional outcomes, quality of life, and survival. In my opinion, pharmaceutical companies such as Sarepta Therapeutics should communicate these results much more openly, rather than relying primarily on promotional narratives and expectations. Learn More: Biomarkers in Duchenne
What is your view on micro-dystrophin production with Elevidys?
Some studies have reported microdystrophin levels reaching up to 17.5% of the normal dystrophin levels observed in healthy individuals without DMD.
However, I do not believe this data alone is meaningful, because we still do not know how this translates into clinical benefit.
If Elevidys could produce 100% normal dystrophin, then perhaps a $3 million price tag might be considered reasonable in the pharmaceutical industry. But even Sarepta acknowledges that Elevidys is not a cure.
What actions have you taken to get more information from Sarepta and Roche?
As DMDWarrioR’s Turkish representative, I sent multiple detailed emails to Sarepta. I asked when Roche would apply for a license in Turkiye.
Sarepta responded only after my fifth email, stating they would consult Roche and get back to us.
Meanwhile, we attempted to schedule a meeting with Roche Turkiye but were unsuccessful.
Ultimately, we received no meaningful response or follow-up.
What are the most important unanswered questions in your mind?
The most critical questions are:
• How long will this treatment extend my child’s life?
• Why does Elevidys cost $3 million?
• What measurable improvements occur in CK, AST, and ALT levels?
• Why are licensing applications not being made in all countries?
• Why is there a lack of communication with patient communities like DMDWarrioR?
What do you think about concerns raised by the Chilean Ministry of Health?
I agree with their concerns. Sarepta must share all available data transparently and clearly explain the rationale behind the $3 million price.
What were the reasons behind the EMA’s decision not to approve Elevidys?
The primary reason behind the European Medicines Agency’s decision was the lack of sufficiently robust and convincing clinical evidence. In particular, the available data did not demonstrate a statistically significant or clinically meaningful improvement in functional outcomes, such as NSAA (North Star Ambulatory Assessment) scores. Read More: Why CK, AST, and ALT Data Matter More Than Videos in Duchenne
Additionally, reductions in creatine kinase (CK) levels were not observed at levels that would indicate a substantial slowing of disease progression. Taken together, these limitations raised concerns about whether the therapy provides a meaningful clinical benefit, which likely influenced the European Medicines Agency (EMA)’s decision not to grant approval.
How do you evaluate other ongoing clinical trials for DMD?
In my opinion, many of them may prove to be more effective and more affordable than Elevidys.
Some studies are already reporting higher levels of micro-dystrophin, and others claim progress toward producing full-length dystrophin. Learn More: New Gene Therapies for Duchenne
Are there promising developments in DMD research globally?
Yes. There are significant studies underway in countries such as Turkiye, Russia, China, France, Canada, and Japan.
We expect more news about these developments in the near future.
Is there ongoing DMD research in Türkiye?
Yes, absolutely. Turkiye has highly valuable researchers working in this field. Their work is progressing rapidly, and we are closely monitoring developments and staying in contact with them.
It is possible that a major breakthrough could come from Turkiye.
Hospitals in the Middle East, such as those in Dubai and Qatar, frequently advertise Elevidys. What are your thoughts on this?
In my view, these hospitals function primarily as treatment centers that administer Elevidys, rather than institutions that develop or modify the therapy itself. While they may have a solid understanding of genetics, they are not involved in creating or advancing gene therapies. Their role is largely limited to delivering a product developed by companies such as Sarepta Therapeutics.
I also believe there is a growing competition among hospitals to attract patients, particularly in high-cost treatments like gene therapy. This raises concerns that financial incentives may play a significant role, especially in conditions such as Spinal muscular atrophy and Duchenne muscular dystrophy, where families are often desperate for treatment options.
Another important concern is clinical preparedness for managing potential side effects. Gene therapies can carry serious risks, and I am not fully convinced that every center offering Elevidys is equally equipped to handle severe complications that may arise after treatment. This uncertainty makes it even more critical for families to carefully evaluate where and how such therapies are administered.
Overall, while access to advanced treatments is important, transparency, medical accountability, and patient safety should remain the top priorities.
What message would you like to share with families affected by DMD?
There is ongoing progress worldwide, and new developments are emerging. Families should remain informed and hopeful, while also continuing to advocate for transparency, accessibility, and fair pricing in treatments.
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Closing Note
We thank DMDWarrioR’s Turkish representative for sharing these candid insights. We hope that Elevidys and future therapies become more accessible, affordable, and transparently evaluated for all patients worldwide.
