A Guide to Duchenne Muscular Dystrophy Treatments and Ongoing Research

This article was updated in September 2025 by Andrea Enguita Marruedo, PhD (Medical Content Writer at myTomorrows) to incorporate new insights and reflect advancements in Duchenne Muscular Dystrophy treatments since its initial publication in 2021.

It is estimated that every year, about 1 in 3,500 to 5,000 boys worldwide is born with Duchenne Muscular dystrophy (DMD). ¹ This rare genetic condition causes muscles to get weaker over time. This can make it harder to walk, climb stairs, and perform daily activities. As the disease progresses, it can also affect the heart and lungs.

There is currently no cure for Duchenne. However, some treatments can help with symptoms and improve quality of life. This blog explores the treatments currently approved in the US and the investigational therapies being studied in laboratories or clinical trials. It also explains how these treatments work and whether they are also approved in Europe or other places.

Our goal is to give a clear picture of the Duchenne Muscular Dystrophy (DMD) treatment landscape, from today’s available treatments to the progress being made in research. It is intended for individuals living with the condition, those involved in their care, or anyone seeking to learn more.

What is Duchenne Muscular Dystrophy (DMD)?

Duchenne Muscular Dystrophy (DMD) is a rare genetic condition that progresses over time. It is caused by mutations (changes in the DNA) in a gene called dystrophin. This gene is responsible for producing the dystrophin protein, which helps keep muscles strong and healthy. ¹

This protein works like a shock absorber in muscle cells. It protects the tiny strands inside muscles (known as muscle fibers) from damage when muscles tighten and relax (called muscle contractions). Without enough dystrophin, the muscles are more likely to get injured during everyday activities. ¹

When the muscles are damaged, the body tries to repair them. Over time, these repair cycles become less effective. This leads to weaker muscles and scarring (fibrosis). Eventually, it can also cause a loss of muscle function.¹

Looking for more information about what causes Duchenne, how it’s diagnosed, and the signs to look for? Read our blog ‘Living with Duchenne Muscular Dystrophy: Treatments and Care Essentials’.

Are there any treatments for Duchenne Muscular Dystrophy (DMD)?

There is currently no cure for Duchenne Muscular Dystrophy (DMD). However, there are treatments that can help with symptoms and improve quality of life.

The most common treatment available for Duchenne is a type of medicines called corticosteroids.

In recent years, other treatments, including genetic therapies, have been approved in the US. Other possible treatments are still being tested in research studies called clinical trials. These studies check if a treatment is safe and effective. ^{2, 3, 4}

This article looks at both approved treatments and those still in research.

A full list of medicines and their approval status in the US and Europe is shown in Table 1.

Corticosteroid Treatments for Duchenne Muscular Dystrophy

In Duchenne Muscular Dystrophy (DMD), the body cannot make enough dystrophin. Without it, muscles are hurt more easily and can swell. This swelling, called inflammation, can cause more muscle damage. ⁵

Corticosteroids are medicines that lower inflammation. They can also help slow down muscle weakness and help the heart and lungs work better for longer. Common corticosteroids used for Duchenne are prednisone, prednisolone and deflazacort. ^{6, 7, 8, 9, 10}

Another corticosteroid that may be available in some countries is called vamorolone. In 2023, it was approved to treat DMD by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). These are the organizations that review and approve new medicines in the US and Europe. ¹¹

Vamorolone was designed to work like older corticosteroids but may cause fewer side effects. More research is needed to know how it compares to other corticosteroids over time. This work is part of a broader effort by scientists to improve medicines, aiming to maintain their benefits, reduce side effects, and make them more manageable for patients in the long term. ^{2, 12, 13}

Other experimental therapies that reduce inflammation are also being tested in clinical trials.

Non-steroidal Treatments for Duchenne

Inside each cell, DNA is packed very tightly. For some genes to work and make proteins, the DNA needs to be more open.

HDACs, or histone deacetylases, are special proteins that wrap DNA more tightly. This can stop some genes from switching on. In Duchenne Muscular Dystrophy (DMD), too much HDAC activity can block the activation of genes that help muscles repair and control inflammation. This can slow muscle healing, keep inflammation going, and cause scar tissue (fibrosis) to build up in the muscles. ¹⁴

Histone deacetylase inhibitors, called HDAC inhibitors, are a type of drug that block HDACs. This helps open up the DNA structure so certain genes can be turned on. In DMD, HDAC inhibitors may help activate genes that help repair and rebuild muscle. They may also help reduce inflammation and fibrosis in the muscles. 2, 15, 16

One HDAC inhibitor, givinostat, has been approved to treat Duchenne by the U.S. Food and Drug Administration (FDA), the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom and the European Medicines Agency (EMA) in Europe.^{17, 18, 19}

Overview of Genetic Therapies for Duchenne Muscular Dystrophy

These therapies aim to correct the underlying genetic cause of the disease. Each of the therapies is designed to target a specific type of mutation in the dystrophin gene. Consequently, they only work for a small subset of patients who possess that mutation. ³

Gene Delivery for Duchenne

Gene delivery works by introducing a healthy gene into the muscle cells, with the goal of replacing the faulty gene that causes the disease. ^{20, 21, 22}

This approach normally uses a carrier (called a vector)  to transport the gene into the cells, which is usually an inactive virus, with the most commonly used being Adeno-Associated Virus (AVV). Scientists modify these viruses in the lab so they won’t cause an infection. ^{20, 21, 22} However, there may still be risks associated to this approach, as some people may have reactions from their immune system, or experience side effects such as liver problems, nerve damage, or other complications. ^{23, 24}

Because the full dystrophin gene is too large to fit inside the virus, researchers use a shorter version called micro-dystrophin. This version produces a smaller protein that can still help protect and repair muscles ^{25, 26}.Elevidys (delandistrogene moxeparvovec-rokl) is the first approved gene therapy for Duchenne Muscular Dystrophy (DMD) in some parts of the world. ^{25, 26}

It is currently available for children with Duchenne who can walk, provided they meet other medical requirements that have been set specifically in each country. Approvals include:

• United States – Approved by the Food and Drug Administration (FDA) for children aged 4 years and older who can walk. ^{25, 27, 28}
• Japan – Approved in April 2025 by the Ministry of Health, Labour, and Welfare (MHLW) for children aged 3 to under 8 years. ²⁹
• Qatar, Kuwait, UAE, Oman, Bahrain, Brazil, and Israel – Approval details vary by country and depend on local regulations. ^{30, 31}

At the time of writing, no other gene therapy has been approved. The European Medicines Agency (EMA) is currently reviewing this product and their decision is pending, which means it is not available as part of regular care. ^{32, 33}

In the United States, Elevidys was made available for children with Duchenne aged 4 and older who cannot walk through the FDA’s accelerated approval pathway, which allows earlier access while more studies are conducted to further investigate the potential risks and benefit. ^{25, 27, 28} However, this approval was paused after two individuals suffered from fatal liver failure.  This pause affects both clinical trials and regular treatment. ^{30, 34, 35}

Other Gene Delivery Therapies Under Investigation

Other gene delivery therapies are being tested in clinical trials. All of them use a smaller version of the dystrophin gene called micro-dystrophin.

These therapies are investigational and are not available as part of regular medical care. Access is generally limited to participation in approved research studies, which are designed to determine whether they are safe and effective.

The approaches differ in several ways, including:

• The type of Adeno-Associated Virus (AAV) used to carry the gene.
• The way the shortened dystrophin gene is built. For example, which parts of the original gene are kept or removed.
• The medicines used to help control the immune system’s response.

These trials are at different stages of testing and may focus on different groups of people. ^{36, 37, 38, 39, 40, 41, 42, 43}

Exon Skipping for Duchenne

Exons are small sections of DNA that act like puzzle pieces, each carrying instructions for part of a protein. When put together, they help form a complete, working protein.

In some cases of Duchenne Muscular Dystrophy (DMD), some of these pieces are missing or deleted from the gene. This prevents the pieces from fitting together properly, resulting in a faulty protein that doesn’t work correctly.

Exon skipping uses small pieces of synthetic DNA to “skip over” the faulty part of the gene in the dystrophin gene when the body is making the protein. It works in this way:

• The small pieces of synthetic DNA hide the faulty part of the gene.
• The body then ignores these pieces and connects the remaining exons.
• As a result, the cell can make a shorter dystrophin protein. ⁴⁴

The shorter protein can still work, but not perfectly. Exon skipping creates a scenario similar to Becker Muscular Dystrophy (BMD). In Becker, some exons are missing, but the rest can still join to make a shorter, partly working protein.⁴⁴

At the time of writing, four exon-skipping therapies are approved by the FDA for Duchenne in the U.S. for individuals with certain mutations that are amenable to exon skipping: Amondys 45 (casimersen), Exondys 51(eteplirsen), Viltepso (viltolarsen) and Vyondys 53 (golodirsen). ^{44, 45} They were approved under the FDA’s “accelerated approval” program, which means more studies are still running to learn how well they work.

Viltepso is also approved in Japan by the Pharmaceuticals and Medical Devices Agency (PMDA). ⁴⁶

These treatments are not approved in other countries outside the U.S. and Japan.

Investigational Genetic Therapies for Duchenne Muscular Dystrophy

Other genetic approaches are also being studied in clinical trials and earlier lab research. These therapies are investigational and are not available as part of regular medical care. Access is generally limited to participation in approved research studies, which are designed to determine whether they are safe and effective. These include other forms of exon skipping, different types of gene therapy, and other strategies such as:

Nonsense Mutation Readthrough for Duchenne

In some cases, Duchenne Muscular Dystrophy (DMD) is caused by a nonsense mutation. This type of mutation puts a “stop signal” in the gene too early. This causes the production of dystrophin to stop too soon, creating an unfinished protein that cannot work. ^{47, 48}

Nonsense mutation readthrough therapy uses a small molecule to help the cell ignore this stop signal. This allows the cell to make the full dystrophin protein. ⁴

One example is ataluren (Translarna). It was once approved in Europe, but the approval was later withdrawn. The EMA’s human medicines committee (CHMP) said there was not enough proof that it was effective. In the U.S., ataluren is not approved, but at the time of writing, the company has asked the FDA to review it again. ^{49, 50, 51, 52, 53, 54}

Gene Editing for Duchenne

Gene editing for Duchenne Muscular Dystrophy (DMD) works by finding a specific DNA sequence and modifying it. The most studied method is the CRISPR-Cas system. CRISPR is a system originally found in bacteria that helps them fight viruses

This gene editing system works in some basic steps:

• A guide molecule directs the system to the exact spot in the DNA that needs to be modified.
• A special protein acts like scissors and cuts the DNA at that spot.
• The cell then tries to fix the cut using its natural repair system.
• During this repair, the faulty DNA in the dystrophin gene may be removed, fix or replaced with a working copy. ^{55, 21}

Currently, there is one clinical trial taking place in China for Duchenne that is using this technique, while other studies are still in the preclinical or laboratory phase. ⁵⁶

Utrophin Modulators for Duchenne

Utrophin is a protein that the body makes on its own. This protein is very similar to dystrophin.

Dystrophin is normally found all around the outside layer of muscle cells, which is called the muscle cell membrane. Utrophin is usually found only in certain spots, like where muscles attach to nerves or tendons.

Researchers are studying ways to increase utrophin levels across the entire muscle membrane. This could allow utrophin to do the job of dystrophin when dystrophin is missing, like in Duchenne Muscular Dystrophy (DMD). ^{2, 57, 58}

Since this approach doesn’t depend on fixing one specific gene problem, it could potentially help anyone who has Duchenne.

This approach is currently being investigated in early clinical trials and preclinical studies.²

Stem Cell Therapy for Duchenne

Stem cells are special cells in the body. They can turn into different types of cells, like muscle cells.​ ⁵⁹

Scientists can take stem cells from a person and grow more of them in a lab. In stem cell therapy for Duchenne Muscular Dystrophy (DMD), these cells are put back into the body. In the muscles, they may help replace damaged muscle cells and help the muscle work better.^{59, 60, 61}

This therapy is currently being investigated in clinical trials. It is not available for regular medical care.

Other Investigational Therapies for Duchenne Muscular Dystrophy

Scientists are also studying other types of treatments for Duchenne Muscular Dystrophy (DMD). They include:

• Calcium balance regulators: Healthy muscles keep the right amount of calcium inside their cells. Calcium helps muscles tighten and relax (muscle contraction and relaxation). In Duchenne, weak muscle cell walls let in too much calcium. This extra calcium causes swelling, scarring, and more muscle damage. Researchers are testing treatments that try to limit calcium entry into muscle cells, lessen the harm it causes, or help fix and strengthen the muscle cell membrane. ²
• Anti-oxidative stress drugs: In Duchenne, muscle cells can be damaged by a buildup of harmful molecules. These are called reactive oxygen species. Scientists are developing treatments that aim to lower these molecules and protect muscle cells. Many of these treatments use antioxidants. ²
• Anti-fibrotic agents: These therapies aim to reduce or prevent fibrosis – the buildup of scar tissue in muscles – which is a contributor to muscle weakness and stiffness in Duchenne. The therapies aim to block the signals in the body that lead to fibrosis, helping preserve healthy muscle tissue for longer and potentially improving muscle function.^{2, 62, 63}

Conclusions

Duchenne Muscular Dystrophy (DMD) is a genetic condition that gets worse over time. It causes muscles to get weaker and smaller. There is no cure at the moment. Current approved treatments aim to slow the disease, keep muscles healthier, and improve quality of life.

Therapies such as corticosteroids (the standard of care in the U.S. and other regions) or histone deacetylase inhibitors (approved only in the U.S) may help manage symptoms and keep muscles working longer.

Genetic therapies aim to restore or compensate for the lack of dystrophin protein. A few of these gene treatments are approved in the U.S., but they are not yet approved in Europe or are still being reviewed.

Other approaches are still being studied in labs or in clinical trials. One is utrophin modulation, which tries to raise levels of utrophin, a protein similar to dystrophin. Another is stem cell therapy, which uses stem cells. The aim is for these cells to help repair or replace damaged muscle.