What treatments are available for Duchenne muscular dystrophy?

Treatments for Duchenne muscular dystrophy (DMD) can be divided into two main groups:

Medications

Drugs that help slow the loss of muscle function and treat the symptoms of DMD.1,2

Supportive care

Manages the complications of DMD and helps improve a person’s quality of life. This includes things like physiotherapy, heart monitoring and breathing support.1,2


DMD medications: what are the options?

While there is currently no cure for DMD, there are medications that can help protect the muscles and ease symptoms. Different types of medications aim to treat the different symptoms of DMD:1–4

Drugs that help reduce inflammation in muscle cells1–4
These are the most commonly used medications for DMD. They are used to help maintain muscle strength for as long as possible.

They may also keep the heart and lungs strong for longer, as well as delaying the development of scoliosis.

Drugs that help muscle cells produce dystrophin4
DMD is caused by a mutation in the gene that codes for dystrophin – a protein that helps to keep muscles working properly. As a result, the body cannot make dystrophin.
 
Medications that target the underlying cause of the disease allow the body to make a working form of the dystrophin protein.
 
These currently available medicines are only suitable for some patients with DMD, due to the way in which they work. Therefore, an understanding of the specific mutation causing DMD may help to determine the right management pathway. This is done through genetic testing and should be completed as early as possible.

Potential new treatment strategies are emerging, some of which are in clinical trials. You should talk to your child’s doctor about treatment options because they are best placed to advise you on medical matters.

Heart medications1
People with DMD may develop a heart problem called cardiomyopathy, which reduces the heart’s ability to pump blood around the body. Eventually, heart failure may develop.

This can be treated with several different medications.

Bone medications1
People with DMD often have weak bones and may develop a condition called osteoporosis. This makes the bones fragile and more likely to break.

Osteoporosis can develop for a number of reasons. Muscle weakness and the use of certain medications contribute to bone weakness.

Bone strength can be increased.1,2,4 Please ask your doctor for more information.

Vaccinations1–4
People with DMD may be offered immunisation with influenza (flu) and pneumococcal (pneumonia) vaccines, to help prevent chest infections.


DMD supportive care: what are the options?

Managing DMD takes more than just medication.1
Because DMD weakens muscles throughout the entire body, it has a variety of effects on different body systems. This makes people with the disorder more susceptible to complications.1
The best way to manage DMD involves a combination of approaches and a team of different specialist doctors, nurses and healthcare professionals.1

Muscle health3

Because DMD causes the muscles in the body to become weak and damaged, maintaining muscle strength and function is a key part of care.

Physiotherapists, occupational therapists and other rehabilitation specialists play a major role in the care of people with DMD. They work on stretching out the muscles and keeping joints as flexible as possible.

As DMD progresses, a scooter, stroller or wheelchair may be needed to help your child or young person get around and be more independent.

Diet3

DMD affects all muscles in the body, including the muscles that help control eating, swallowing and digestion. This can cause constipation and gastroesophageal reflux, as well as make chewing and swallowing difficult.

A healthy diet and healthy weight are especially important for people with DMD. A dietitian will help make sure that your child or young person receives a well-balanced diet and the right amount of nutrients, supplements and fluids. A gastroenterologist will also be able to help with any digestive problems.

Bone health1

People with DMD often develop a condition called osteoporosis. This makes the bones fragile and more likely to break. As back muscles become weaker, the spine may also begin to curve – this is known as scoliosis.

Radiographs or X-rays are used to detect the earliest signs of bone weakness, and medication may be used to treat osteoporosis. Surgery or casting may also be offered in the case of broken bones (fractures) or scoliosis.

Breathing support1,3

The muscles used for breathing are also affected by DMD. As the disease progresses, these muscles get weaker and people with DMD start to have trouble breathing and coughing.

Breathing problems can be reduced by various machines that help the lungs to breathe (ventilators) and cough (cough assist machines).

Heart health1

DMD affects all of the body’s muscles – including the heart. That is why your child or young person’s heart will be checked often by a cardiologist.

The cardiologist may suggest using medication if the heart is damaged – a condition called cardiomyopathy – or to help reduce future damage.

Vaccinations1–4

Because people with DMD often have trouble coughing, chest infections are more common. This is because coughing helps clear mucus and germs from the respiratory tract.

Influenza (flu) and pneumococcal (pneumonia) vaccines may be offered. These vaccinations help prevent chest infections from happening in the first place.

Learning2,3

Dystrophin proteins have been found in the brain. The role that they play is unclear, but this could help explain why problems with learning, speech and behaviour tend to be more common in people with DMD.

A number of different mental health professionals will be able to provide support, depending on the person’s individual needs. Schools will also be able to adapt to both physical and learning needs.

As DMD is a progressive disease, meaning the symptoms become more apparent over time, it is important to get your child the right treatments and management as soon as possible. Talk to your healthcare professional about the options

1. Birnkrant DJ, et al. Lancet Neurol. 2018;17:347–361 [Part 2].
2. Birnkrant DJ, et al. Lancet Neurol. 2018;17:251–267 [Part 1].
3. Bushby K, et al. Lancet Neurol. 2010;9:77–93.
4. Mah JK. Neuropsychiatr Dis Treat. 2016;12:1795–1807.

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