Registered charity
number 1146355

Treat NMD

What is the treatment for Duchenne
muscular dystrophy?


There is presently no cure for DMD at present. However, carmela.JPG
treatments can help. Here is is an outline of the treatment
you/your child may be offered. The treatment is described
here according to age group, although there will be some
overlap and the ages given are only a rough guide.


Preschool age

Usually, at this stage, your child will be well and not need
much treatment. What you will usually be offered is:

Information about DMD. You may wish to be in touch with
patient support groups or other families with DMD. Referral
to a specialist team (for example, a paediatrician or neurologist,
physiotherapist and a specialist nurse) so that your child's
health can be monitored.Advice about the right level of exercise
for your child.Genetic advice for the family. You may wish to
have tests to see whether anyone else in the family has the DMD
gene. This may be important to families who are thinking of
having more children.


Age 5-8 years

At this age, some support may be needed for the legs and ankles. For example, using night time ankle splints.

Treatment with medication called 'corticosteroids' (or 'steroids') can help to maintain the child's muscle strength. This involves taking medication such as prednisolone or deflazacort as a long-term treatment, either continuously or in repeated courses. Steroid medication can have side-effects, so the pros and cons of this treatment need to be weighed up, and the treatment needs to be monitored for side-effects.


8 years-late teenage years

At some time after the age of 8 years, the child's leg muscles become significantly weaker. Walking gradually gets more difficult, and a wheelchair is needed. The age at which this happens varies from person to person. Often it is around age 9-11 years, although with corticosteroid treatment, some boys can walk for longer.

After the child starts needing a wheelchair, this is also the time that complications tend to begin, so it is important to monitor the childs health and to treat any complications early (see below). Your child will need regular check-ups. This may involve different specialists, for example, heart and lung checks, orthopaedic care for bones and joints, physiotherapists, and dieticians.

Practical support and equipment will be needed at this stage, for example, wheelchairs and adaptations to the child's home and school. Occupational therapists can advise about this. Various services can assist with equipment, care, holidays and breaks. There is usually provision from local health and social services. Also, various charities which can assist with equipment, holidays and other forms of care.

Counselling and emotional support for you and/or your child may be helpful.


Late teenage years-twenties

At this stage, muscle weakness becomes more problematic. Increasing help and adaptations are needed. Complications such as chest infections are likely to increase, so more medical monitoring and treatment are required.

 

What are the complications of Duchenne muscular dystrophy, and how are they treated?


Anaesthetics

People with DMD need extra care if they have a general anaesthetic. Certain anaesthetic medicines can cause a harmful reaction for people who have DMD. Also, extra care for the chest and breathing is needed. It is important to have a pre-operative assessment and a senior anaesthetist providing the anaesthetic care. Always tell doctors and nurses that you/your child has DMD and on steroids if relevant.


Osteoporosis

People with DMD may develop osteoporosis (thinning of the bones). This is due to lack of mobility and also to steroid treatment. It is important to prevent osteoporosis as far as possible. A good intake of vitamin D and calcium helps keep bones strong. These can be obtained from certain foods or from vitamin supplements, and by action of sunshine on the skin (which makes natural vitamin D).

Sometimes a blood test to check your vitamin D levels is advised, and vitamin D supplements may be offered.

Osteoporosis which is problematic can also be treated with certain medications, for example with 'bisphosphonates'.


Joint and spinal complications

Muscle weakness can result in some joints becoming tight - this is called a 'contracture'. In DMD, it is often the ankle joint and Achilles' tendon which become tight. This can be treated either using orthotic devices (splints or casts) or by surgical release of the tendon.

Curvature of the spine (scoliosis) can occur due to muscle weakness. Usually this happens sometime after the child needs a wheelchair. The scoliosis can cause discomfort and is not helpful for posture and breathing. Treatments which can help are a spinal brace, or surgery to the spine.


Nutrition and digestion

Some children with DMD are prone to being overweight, especially if taking steroid treatment. Teenagers and adults with DMD may be underweight, due to loss of muscle bulk. Dietary advice can be helpful in these situations.

Constipation can be a symptom for anyone who is not mobile. This can be treated with laxatives and a high fibre diet.

In the later stages of DMD (as a young adult and older), people with DMD may have difficulty with chewing and swallowing food. They may need a careful assessment and nutritional advice or supplements. If the problem is severe, then a 'gastrostomy' (a feeding tube into the stomach) may be needed.

 

Chest and breathing complications

During the teenage years, the breathing muscles weaken, causing shallow breathing and a less effective cough mechanism. This can lead to chest infections, because mucus and bacteria are not cleared so easily from the chest. It is important to treat chest infections promptly, for example by getting medical advice early and taking antibiotics where needed. Also, you will be offered immunisation with influenza and pneumococcal vaccines, to help prevent chest infections. Physiotherapy can help to clear mucus from the chest.

As the breathing muscles get weaker, oxygen levels in the blood may be reduced, more so while sleeping. Because this develops gradually, the symptoms may not be obvious. Possible symptoms are tiredness, irritability, morning headaches, night time waking and vivid dreams.

Breathing problems can be helped in various ways. There are techniques to assist breathing and coughing which can be taught by a physiotherapist. Also, various types of equipment can be used to assist breathing. Often, what helps is a method called 'non-invasive ventilation'. This uses a mask worn over the mouth or nose and delivers air which is pressurised. The extra air pressure compensates for the breathing muscles' weakness. Non-invasive ventilation can often be used in a way which does not interfere too much with other activities, for example, it may only be needed at night or intermittently. The equipment can be portable.

There are various other methods to help improve breathing and oxygen levels. The aim is to find the one most suited to each individual.

It is helpful if breathing problems are detected and treated early; so patients with DMD will usually be offered regular 'lung function tests' once they start to have significant muscle weakness. These are simple tests which can monitor the strength of the breathing muscles and the level of oxygen in the blood.

In the later stages of DMD, the muscle weakness progresses, and more support will be needed for breathing. This type of treatment has improved over recent years, which has helped to improve the quality of life and outlook people with DMD.


Cardiac (heart) complications

Teenagers and adults with DMD may develop a heart problem called cardiomyopathy, which means weakness of the heart muscle. With DMD, the cardiomyopathy does not usually cause much in the way of symptoms. Possible symptoms are tiredness, leg swelling, shortness of breath or an irregular heart beat. Cardiomyopathy can be helped by medications such as Ace Inhibitors and beta Blockers. This treatment seems to work best if started at an early stage, before symptoms are noticed. So people with DMD are usually offered regular heart check-ups, starting from early childhood. The check-ups usually involve an ECG (recording of the heartbeat) and 'echocardiogram' or 'echo', which is an ultrasound of the heart.

Support & Advice

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Supplements and medications

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Adaptations

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Educational needs and benefits

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Wheelchairs and equipment

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