At present there is no cure for muscular dystrophy (MD), but a variety of treatments can help to manage the condition.
As different types of MD can cause quite specific problems, the treatment that you receive will be tailored to your needs. As your symptoms develop, the healthcare professionals who are treating you should advise you about the options.
New research is investigating possible future treatments. Improved genetic testing can help if you are concerned about passing the condition on to your children. Read more about genetic testing.
Mobility assistance
As MD progresses it weakens your muscles and you gradually begin to lose mobility and strength. These physical problems can be helped with the following:
- exercise keeps the muscles working in a good condition, because a lack of activity can make the condition worse
- physiotherapy can be useful for maintaining muscle strength and also the length of tendons, which is important for preserving flexibility and preventing stiff joints
- physical aids, such as a wheelchair, leg braces or crutches, can help you to maintain standing and stay mobile
Support groups
Muscular dystrophy can affect you emotionally as well as physically. Support groups and organisations may help you to understand and come to terms with your condition better. They can also provide useful advice and support for those who care for people with MD.
There are several national charities such as the Muscular Dystrophy Campaign that offer support for people affected by MD. Or you can ask your GP or other healthcare professional treating you about local support groups.
Steroid medication
In people with Duchenne MD, corticosteroid medication has been shown to improve muscle strength and function for six months to two years. For some people, it can improve:
- the time it takes to stand up from the floor
- walking speed
- the ability to climb stairs
- the ability to lift weights
Some side effects, such as weight gain and excessive hair growth, are associated with using steroid medication.
Surgery
In some severe cases of MD, surgery may be necessary to correct postural deformities. For example, if you or your child has Duchenne MD, you (or they) may develop scoliosis, which is where the spine curves to the side.
Surgery can correct the scoliosis, although there have been no trials to evaluate its effectiveness. Surgery is aimed at:
- halting the progression of MD
- improving cosmetic appearance
- maintaining movement and function in the upper limbs and chest
- increasing life expectancy
If you or your child may benefit from having surgery you will be referred to a specialist to discuss the procedure and the risks involved.
Other kinds of surgery may be used to treat specific symptoms. For example, if you have ptosis (droopy eyelids) caused by oculopharyngeal MD, surgery can successfully lift the eyelids away from your eyes and restore your vision. Several treatments are also available for dysphagia (difficulty swallowing).
Treating heart complications
Some types of MD can affect the heart muscles and the muscles used for breathing. When the condition has progressed to this stage it can become life-threatening.
It is important that your heart function is assessed regularly once MD has been diagnosed. For Duchenne and Becker MD, a magnetic resonance imaging (MRI) scan or an echocardiogram (ECG) should be carried out about once a year to check for any damage.
If any damage to your heart is detected you may be referred to a cardiologist (a heart specialist) for tests every three months. You may also be prescribed medication to treat your heart problems. ACE inhibitors relax your arteries, making it easier for your heart to pump blood around your body. Beta-blockers can control irregular heart beats (arrythmias) and make it easier for your heart to work.
In some cases of myotonic MD, a pacemaker may be fitted to correct an irregular heartbeat. A pacemaker is a small, battery-operated device that can be implanted into your chest to regulate your heartbeat.
New research
New ideas for MD treatments are currently being developed. Browse clinical trials for muscular dystrophy. Your GP or the healthcare professional treating you may also know of any recent developments that might benefit you.
Speak to your GP if you are interested in taking part in a clinical trial (a form of research that tests one treatment against another).
Some examples of current research ideas are explained below.
Stem cell research
Stem cells are cells that are at an early stage of development. This means they have the ability to turn into any type of cell in the body. Some research is currently focusing on whether stem cells can be turned into muscle cells and used to regenerate damaged muscle tissue.
Exon skipping
In the future, ‘exon skipping’ may be a way of treating Duchenne MD. Exons are sections of genetic coding (DNA) that contain information for proteins. In Duchenne and Becker MD, some of the exons are missing or duplicated, which can interfere with the dystrophin protein being produced.
Researchers are currently investigating ways of ‘skipping’ additional exons in the dystrophin gene. This could mean that more dystrophin would be produced, reducing the severity of MD symptoms.
The trials at the moment are focused on treatment that would apply to Duchenne MD but it may become applicable to Becker MD in the future.
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