ELEVIDYS for Duchenne: New Data from the 2026 World Muscle Society Congress
Written by the Muscular Dystrophy Pakistan (MDP) content team. Based on reports from Parent Project Muscular Dystrophy, NeurologyLive and Sarepta Therapeutics (see sources below). Published: October 17, 2026.
The 31st Congress of the World Muscle Society, held in Hiroshima, Japan, brought new numbers on ELEVIDYS (delandistrogene moxeparvovec), the gene therapy for Duchenne muscular dystrophy made by Sarepta Therapeutics. The data were presented on September 30, 2026. This article explains what the new results show, what they do not show, what the safety label says, what the treatment costs, and what it means for families in Pakistan. For background on Duchenne itself, read our complete guide: Duchenne Muscular Dystrophy (DMD): Symptoms, Causes, Life Expectancy and Treatment.
Quick facts
- ELEVIDYS is a one-time intravenous gene therapy that delivers a shortened, working copy of the dystrophin gene (micro-dystrophin) to muscle cells.
- New data compared 25 walking boys aged 8 to 12 with 99 matched external controls over 2 years and found better motor function in the treated group.
- A second analysis in 9 children aged 2 to under 3 showed micro-dystrophin in muscle but did not establish a functional benefit.
- Since November 2025, the US label is limited to ambulatory patients aged 4 and older and carries a boxed warning for acute serious liver injury and liver failure.
- The US list price is about US$3.2 million for one infusion. To our knowledge, it is not available in Pakistan.
What Is ELEVIDYS?
In Duchenne, the DMD gene is too faulty to make working dystrophin. ELEVIDYS uses a harmless virus (an AAV vector) as a delivery vehicle to carry a shortened version of the gene, called micro-dystrophin, into muscle cells, so that the muscle can make a small, working form of the protein. It is given once, as an infusion into a vein, and it was approved by the US FDA in 2023. It is not a cure, and its long-term effects are still being studied.
What Did the New Data Show?
Boys aged 8 to 12
Investigators analysed 25 walking boys aged 8 to 12 from the EMBARK and ENDEAVOR studies, and compared them with 99 well-matched boys from natural history data, who were not treated. After two years, the treated boys did better on three measures of movement:
| Measure | Difference after 2 years | What it measures |
|---|---|---|
| North Star Ambulatory Assessment | 3.32 points better | Overall walking and movement skills |
| Time to rise from the floor | 0.041 rises per second faster | Getting up from the floor |
| 10-metre walk/run | 0.195 metres per second faster | Walking and running speed |
Sarepta described the benefits as clinically meaningful and consistent with earlier results in boys treated at ages 4 to 7. An important limit: the comparison group was external, meaning it was not a randomised, concurrent placebo group. Such comparisons are useful, but less strong than a randomised trial.
Children aged 2 to under 3
In 9 young children from the ENDEAVOR and ENVOL studies, researchers measured how much micro-dystrophin was made after treatment. In ENDEAVOR (6 children), the average was about 94% of control levels by Western blot, and in ENVOL (3 children), about 73%. This is a measure of biology, not of how well the children move, and the analysis did not establish a functional benefit in this age group. The researchers say that long-term function is the most meaningful measure of success.
Safety: What the Label Says
- Boxed warning for acute serious liver injury and acute liver failure, including life-threatening and fatal cases. These problems usually begin within 8 weeks of the infusion.
- In 2025, there were fatal liver failure cases in non-ambulatory patients. As a result, the US label was changed in November 2025: the indication was limited to ambulatory patients aged 4 years and older with a confirmed DMD gene mutation, and the non-ambulatory indication was removed.
- The prescribing information calls for liver tests before infusion, steroid treatment before and after, weekly liver tests for the first three months, and staying near an appropriate hospital for at least two months after infusion.
- Other warnings include serious infections, myocarditis (inflammation of the heart muscle), infusion reactions and immune-mediated muscle inflammation.
- ELEVIDYS cannot be used in people with a deletion in exon 8 and/or exon 9 of the DMD gene.
- In the new data, the most common treatment-related side effects were nausea and vomiting.
- Sarepta is running a study of an extra immune-suppressing medicine (sirolimus) to see whether treatment can be made safer for non-walking patients. The outcome is not known yet.
How Much Does It Cost?
The US list price is about US$3.2 million per infusion, which makes it one of the most expensive medicines in the world. This price is for the medicine alone, and does not include hospital stay, liver monitoring, steroids and travel. The treatment was launched in Japan in February 2026 through Chugai Pharmaceutical.
What It Means for Families in Pakistan
- Availability: To our knowledge, ELEVIDYS is not registered or available in Pakistan. A hospital would need DRAP permission to import an unregistered medicine for a named patient, and the infusion needs a specialised centre with experience in gene therapy and liver monitoring.
- Cost: At about US$3.2 million, it is out of reach for almost every family, and no public programme in Pakistan, as far as we know, funds it.
- Eligibility: Even where available, it is for walking boys aged 4 and older with a confirmed DMD gene change, and it is not suitable for everyone. A complete genetic report and a specialist opinion are the first steps.
- Honest expectations: The results are encouraging, but they come from small groups compared with external controls, and the long-term outcome is not yet known. The safety risks are serious.
- Proven care first: Steroids, heart monitoring, breathing care and vaccines remain the foundation for every boy with Duchenne.
What Families Can Do Now
- Get a complete genetic report showing the exact DMD gene change.
- Ask your neurologist about the current options for your child, and whether any clinical trial may suit you in the future.
- Keep the basic care in place: steroids as advised, heart checks, breathing care and vaccines.
- Do not pay brokers or unofficial sellers for any gene therapy or "stem cell" treatment.
- Register with MDP, so that the needs of Duchenne families in Pakistan can be counted and heard.
Frequently Asked Questions
Is ELEVIDYS a cure for Duchenne?
No. It aims to make a small, working form of dystrophin. Studies show better movement compared with untreated boys, but the long-term effect is still being studied.
Can my son with Duchenne get ELEVIDYS?
In the US, it is approved only for walking patients aged 4 and older with a confirmed DMD gene change, excluding those with an exon 8 or 9 deletion. In Pakistan, it is not available to our knowledge.
Why was it removed for non-walking patients?
Fatal acute liver failure cases occurred in non-walking patients in 2025. The FDA changed the label in November 2025, and the company is studying a stronger immune-suppressing regimen.
Is it available anywhere besides the US?
It was launched in Japan in February 2026. Availability in other countries differs, and families should ask their specialist.
Hope With Open Eyes
Gene therapy is one of the most important areas of Duchenne research, and every new data set is worth following. But families deserve both hope and the full picture: promising results, serious risks, a very high price and no access in Pakistan yet. Follow the MDP blog for more research updates.
Sources and Further Reading
- Parent Project Muscular Dystrophy, Catch up on the 31st Congress of the World Muscle Society
- NeurologyLive, ELEVIDYS shows sustained functional differences vs external controls in older ambulatory Duchenne patients
- Sarepta Therapeutics, New data at the 2026 World Muscle Society Congress
- Sarepta Therapeutics, FDA approval of updated ELEVIDYS prescribing information
- CGTLive, Sarepta's Elevidys gets boxed warning for ALI and ALF
- Managed Healthcare Executive, Sarepta adds boxed warning to Elevidys about risk of liver disease
Medical and financial disclaimer: This article is for awareness and education only. It is not medical, legal or financial advice. Treatment details, prices and regulatory status may change. Please discuss any treatment decision with your child's specialist and do not buy gene therapies from unofficial sellers.
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