Treatments for Duchenne muscular dystrophy

Steroids

Treatment with corticosteroids (often referred to as steroids) is recommended as the standard of care in Duchenne. Research and studies have shown that they maintain or slow down the decline of muscle strength and function over a period of time, allowing people with Duchenne to walk and maintain arm function for longer. Over the long term, corticosteroids may also help with upper body and spine strength, as well as working to delay breathing and heart complications.

Corticosteroids are anti-inflammatory drugs. Although the way they work in Duchenne is not fully understood, we know they help to slow down disease progression by reducing inflammation and maintaining muscle strength for longer. However, steroids also have significant side effects, which need to be carefully monitored and appropriately managed.

On average, children who take steroids walk for three years longer than those that don’t.

Steroid treatment has significant side effects on your body, which need to be carefully monitored and appropriately managed. These vary in when they occur, their severity and not every person develops all side effects. Some can be seen as early as within the first six months of starting treatment, whilst others might take longer to develop.

Adrenal insufficiency is a serious side effect of steroids where the body cannot produce enough cortisol during illness and accidents. It can be life threatening and requires careful management. If your child is prescribed steroids, you will be given advice on sick day management and an emergency care plan to initiate in the event of serious illness or accidents.

There are three main corticosteroids that may be used to treat Duchenne: prednisolone, deflazacort or vamorolone. Prednisolone and deflazacort have been used in the treatment of Duchenne since the late 1990s and are often referred to as “classic corticosteroids”. Both have been shown to be effective in the treatment of Duchenne and based on the current evidence, they do not significantly differ in terms of their effect on muscle strength and function. They also have similar side effects. However, some side effects can be seen more frequently with one type of steroid compared to another. 

Vamorolone (also referred to by its brand name Agamree®) is a newer steroid that has been approved in the UK since January 2025 for treatment of people aged 4 years and over with Duchenne. Vamorolone is still a corticosteroid, but it was designed to keep the benefits of classic corticosteroids, whilst reducing some of the side effects. It has been available in the UK on prescription for patients with Duchenne where appropriate, from neuromuscular teams since April 2025.

Other approved treatments

Alongside corticosteroids, there are two approved treatments that may be prescribed to treat patients with Duchenne. Not all treatments are suitable for every patient or for every stage of Duchenne, so speak to your doctor about your/your child's needs.

These approved treatments are:

Givinostat

Givinostat (brand name Duvyzat) is an ‘HDAC inhibitor’. It blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on’ and ‘off’ within cells. It works by targeting pathogenic processes to reduce inflammation and muscle loss. Givinostat is an oral medication taken daily. It can have serious side effects and requires careful monitoring and management and there are several tests required to assess a patient’s suitability for treatment with givinostat as there are a number of exclusionary criteria that can make it unsuitable for some patients.

Givinostat is current being made available to people six years and older in the UK who are ambulant through an Early Access Programme (EAP). The EAP is delivered at the discretion of each hospital and NHS Trust. Not all hospitals and NHS Trusts are delivering the EAP, meaning it is not currently available everywhere. 

Givinostat is currently being assessed by the National Institute for Health and Care Excellence (NICE), which will decide whether to approve the medicine for reimbursement and routine use on the NHS. If approved, it would be made widely available.

Ataluren

Ataluren (brand name is Translarna), was approved by NICE in January 2023 to treat Duchenne in people who are two years and over, and who are ambulant (can walk). Ataluren can only be given to patients who have a specific type of genetic defect called a nonsense mutation, which is approximately 13% of people with Duchenne.

While it is still available in the UK, the European Commission announced in March 2025, the European Commission issued a decision to not renew the conditional marketing authorisation for ataluren in the European Union (EU). 

Other medications and supporting therapies can improve the quality and length of life of those living with Duchenne and ease the symptoms of the disease. 

Cardiac treatments

The heart is a muscle and needs dystrophin to function efficiently. In Duchenne, a lack of dystrophin means that heart muscles weaken over time. Heart cells are replaced by scar and fatty tissue, which leads to a type of heart muscle disease called dilated cardiomyopathy. The age when cardiomyopathy happens in Duchenne can vary from person to person, from early childhood to adulthood. 

Children with Duchenne don’t usually have cardiac symptoms. However, weakening heart muscles are part of the natural progression of the disease. Therefore, early monitoring, preventative treatment, and ongoing management are important for keeping the heart healthy into adulthood.

Steroids and heart medications can slow the decline in heart function. 

There are several different ‘families’ of drugs that protect the heart in Duchenne. Your medical team will discuss benefits and side effects with you and monitor your child closely when they are first introduced. They will also check and adjust dosage according to your child’s weight and age. These medications include the following:

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Angiotensin converting enzyme inhibitors (ACEis or ACE inhibitors) 

These relax the blood vessels, reduce blood pressure and support heart function. ACE inhibitors allow the heart to work more effectively, even when damaged by Duchenne. The names of some ACE inhibitors are: enalapril, lisinopril, perindopril, ramipril – but there are others. ACE inhibitors are the first line of treatment for Duchenne. They should be introduced no later than age 10 to prevent future heart decline, even though heart function will usually still be normal at this age. 

ACE inhibitors are usually well tolerated even by young children. However, rarely they can cause side effects. This can include a persistent dry cough. If side effects occur, most people can be changed to an angiotensin-receptor blocking agent (ARB) medication.

Angiotensin-receptor blockers (ARBs) This group of medications have similar actions to ACE inhibitor drugs but work slightly differently. Your child may be prescribed ARBs if they experience side effects with ACE inhibitors. The names of some ARBs are losartan, irbesartan, candesartan – but there are others.

Angiotensin-receptor blockers (ARBs) 

This group of medications have similar actions to ACE inhibitor drugs but work slightly differently. Your child may be prescribed ARBs if they experience side effects with ACE inhibitors. The names of some ARBs are losartan, irbesartan, candesartan – but there are others.

Beta-blockers (BBs) 

Beta-blockers slow heart rate and reduce blood-pressure by reducing the effects of the hormone adrenaline. They may be prescribed if your child’s heart rate is persistently too fast (sinus tachycardia). The names of some beta-blocking drugs are metoprolol, bisoprolol, carvedilol – but there are others. Beta-blockers can cause cold extremities (toes and fingers) especially in older, less mobile adults with Duchenne.

Mineralocorticoid receptor antagonists (MRAs)

MRAs are a type of diuretic. These lower blood pressure and reduce excessive fluid around the body by increasing the amount of urine (wee) you pass. At more advanced stages of heart weakness, the body retains excessive water, so MRAs help to clear this. However, MRAs also help to prevent scarring in the heart, so can sometimes be prescribed in early stages of Duchenne in combination with an ACE inhibitor too. Two currently available MRAs are spironolactone and eplerenone.

ACE inhibitors (or ARBs) should be prescribed no later than age 10 as a preventative measure to protect the heart in Duchenne, even if the heart seems completely normal on ECHO-scanning. If an ECHO or MRI shows any signs of heart weakness, or your cardiologist recommends it, combinations of heart medication may be prescribed to keep the heart healthy for longer. For example, an ACE inhibitor, an MRA and a BB may all be used together.

Bone Protection

People with Duchenne often have weak bones or reduced bone mineral density, this can be exacerbated by decreased mobility, muscle weakness and the use of steroids. This can lead to the weakening and thinning of bones, called osteoporosis, which makes fractures (broken bones) more likely. 

There is currently no medication that can build up bone in growing children, but the following can help keep bones healthy for longer: 

• Eating a diet rich in calcium (such as dairy, fortified bread and leafy greens). 
• Taking vitamin D supplements as recommended by your doctor. 
• Appropriate physical activity as recommended by a physiotherapist. 
• Boys should be checked for signs of puberty by age 12. If puberty is delayed, testosterone treatment can help to strengthen bones.

Medication for treatment of osteoporosis (such as bisphosphonates) may be prescribed if there is a diagnosis of osteoporosis. Other osteoporosis medications can be prescribed for adults with Duchenne, once they have stopped growing.

Bisphosphonates are medicines that strengthen bones and can reduce the risk of fractures. They are usually given as an infusion via a drip in the arm. The commonly used bisphosphonate infusions are Zoledronate or Pamidronate. Initially, bisphosphonate infusions are usually given every four months (Pamidronate) or every six months (Zoledronate). After a few years, the infusions may be given once a year. 

Bisphosphonates are generally given regularly until the person with Duchenne stops growing.

Physiotherapy and Occupational Therapy

Physiotherapy helps keep muscles strong and flexible, so you can move and do as much as possible. Occupational therapy aims to make everyday tasks possible by making adjustments to fit what you are able to do, and by offering helpful equipment when needed. These two therapies work together to create personalised goals, making sure the care fits your needs and way of life. This team approach can help people with Duchenne stay active and independent in their daily lives for longer.

Physiotherapy and occupational therapy needs will change over time and as Duchenne progresses. 

You can find out more about the type of interventions and support that should be provided at each stage of Duchenne in our Physiotherapy and Occupational Therapy guidelines.

Respiratory Care

In Duchenne, a lack of a protein called dystrophin means that all muscles weaken over time. This includes the respiratory muscles that control breathing and coughing. One of these muscles is the diaphragm. The diaphragm is below the lungs and works to pull air into the lungs. The lungs are not directly affected by Duchenne, but the muscles around them are.

Although it is unusual to see breathing problems until a person with Duchenne is no longer walking independently, it is important to start routine respiratory (breathing) checks from around six years of age.

People with Duchenne who are ambulatory (able to walk) should have checks every 6-12 months. People who are non-ambulatory (not able to walk) should have checks at least every six months. More frequent checks may be required if a person with Duchenne has any symptoms suggesting problems with their chest or breathing muscles – this includes frequent chest infections.

The following steps can help keep the respiratory muscles working well for as long as possible: 

• Follow the recommended care for the heart and bones: both heart muscle weakness and scoliosis (curved spine) can make respiratory muscles weaker

• Prevent respiratory illness by getting vaccines recommended by the government, including an annual flu vaccine 

• Treat chest infections quickly with antibiotics 

• Using high doses of corticosteroids has been shown to slow down changes in the respiratory muscles caused by abnormal dystrophin levels. 

Your child may also be referred for an overnight sleep study to check their breathing patterns through the night. This can be at home or in hospital and is used to check for any sleep disordered breathing, which is often the first indication of the need for additional respiratory support and management for someone with Duchenne. 

As the condition progresses, more specialist respiratory care will be required.

Nutrition

People with Duchenne also need a healthy, well-balanced diet and can take nutritional supplements. This is to support health and manage weight, as weight gain is common in Duchenne due to reduced mobility and the side effects of steroids. We are currently developing nutritional guidelines through Duchenne Care UK to support families and clinicians to ensure the best care and support.

Puberty and testosterone treatment

Puberty is the period when a child’s body develops into an adult. This process often takes about three years. The average age when puberty starts in a boy is approximately 11.5 years although it can range between 9 and 14. People taking corticosteroids are very likely to experience delayed puberty. Puberty may never start or start but not progress normally. 

Steroid medicines used in Duchenne mainly affect the release of the puberty initiating hormones from the pituitary gland (LH and FSH). This affects testosterone production by the testes. Lack of puberty can affect a child with Duchenne’s self-esteem. Boys with Duchenne also have weaker bones with an increased risk of fractures due to the underlying condition and long-term use of steroid medicines. Puberty hormones help to make the bones thicker and stronger. If puberty is delayed or absent, this can further weaken bones.

Your doctor should examine your son for signs of puberty about once a year from around 12 years of age. 

Your doctor may refer your son to a specialist called an endocrinologist to assess his development. This will involve checking the size of his testes. It’s important to explain this to your son before his appointment, so he understands the need for this examination.

The endocrinologist may request blood tests to look at the hormones that control puberty and an x-ray of your son’s hand and wrist to determine growth potential. Testosterone treatment is recommended by 14 years of age, if there are no signs of puberty. It may be considered from 12 years of age if there are no signs of puberty together with concerns about weak bones. 

Treatment can be given by monthly injections or daily gels over two to three years. This mimics the gradual increase in testosterone levels during puberty. Testosterone treatment will lead to an increase in size of the penis and development of pubic and body hair. Your son will look more physically mature, and his voice will deepen with time. His height may also increase. This may help him to feel more confident and similar to his friends. It can also help strengthen bones and stabilise bone density.