How can duchenne muscular dystrophy be treated




















Your GP or the healthcare professional treating you may know of any recent developments in healthcare that may benefit you. Trials are now in progress in the UK and the Netherlands to see if "exon skipping" may be a useful way of treating Duchenne MD. Exons are sections of 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. Current trials are focused on treatment that would apply to Duchenne MD, but it may become applicable to Becker MD in the future. 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. Muscular dystrophy can affect you emotionally as well as physically. Support groups and organisations may help you understand and come to terms with your condition.

They can also provide useful advice and support for people who care for those with MD. You can also ask your GP or other healthcare professional treating you about support groups in your local area. Page last reviewed: 20 July Next review due: 20 July Mobility and breathing assistance As MD progresses, it weakens your muscles and you gradually begin to lose mobility and strength.

These physical problems can be helped with: low-impact exercise , such as swimming physiotherapy , which can be useful for maintaining muscle strength, preserving flexibility and preventing stiff joints physical aids, such as a wheelchair , leg braces or crutches, which can help you stand and stay mobile occupational therapy , which can help maximise or improve your independence by using different techniques, changing your environment and providing any necessary assistive equipment Once the chest muscles become too weak to control breathing properly, you may need machines to assist with your breathing and coughing, particularly while sleeping.

Steroid medicine In people with Duchenne MD, corticosteroid medicine steroids has been shown to improve muscle strength and function for 6 months to 2 years, and slow down the process of muscle weakening. Ataluren Ataluren is a newer medicine that has been developed to treat some children with Duchenne MD aged 5 or older who can still walk.

Read the NICE guidance about the use of ataluren for treating Duchenne MD Creatine supplements Recent research has also shown that a creatine supplement can improve muscle strength in some people with MD, while causing few side effects. Treating swallowing problems People with some types of MD find swallowing increasingly difficult as the condition progresses.

Treating heart complications Some types of MD can affect the heart muscles and the muscles used for breathing. Corrective surgery In some severe cases of MD, surgery may be necessary to correct physical problems that can occur as a result of the condition.

Duchenne muscular dystrophy. Genetic and Rare Diseases Information Center. May 7, Muscular Dystrophy Association. June 13, What Is Duchenne Muscular Dystrophy? Parent Project Muscular Dystrophy. Duchenne Muscular Dystrophy. National Organization for Rare Disorders. Duchenne Muscular Dystrophy in Children. Cedars Sinai. Johns Hopkins Medicine. Muscular dystrophy. Mayo Clinic. January 31, By subscribing you agree to the Terms of Use and Privacy Policy.

Health Topics. If you have questions about treatments for MD, please discuss them with your healthcare provider. MD treatments may include the following. Beginning physical therapy early can help keep muscles flexible and strong. A combination of physical activity and stretching exercises may be recommended for people with MD.

Because the body relies on muscles such as the diaphragm to breathe, weakened muscles from MD may affect breathing. Many people with MD do not realize they have lost respiratory strength until they have difficulty coughing or an infection leads to pneumonia. Shortly after MD is diagnosed, specialists can suggest treatments to prevent or delay respiratory problems.

Eventually, some people with MD may need a ventilator to help them breathe. MD patients who experience weakness in the facial and throat muscles may benefit from speech therapy to teach them how to maximize their muscle strength.

Some methods include slowing the pace of their speech, pausing more between breaths, 3 and using specialized communication equipment. As physical abilities change, occupational therapy can help patients with MD relearn lost motor skills and learn ways to work around weakened muscles.

Occupational therapy also teaches people with MD how to use assistive devices, such as wheelchairs, eating utensils, and personal items, including hair- and toothbrushes. At various times and depending on the form of MD, many people with MD need surgery to treat some of the conditions associated with MD. For instance, people with myotonic MD may need to have a pacemaker installed to treat heart problems or surgery to remove cataracts, a clouding of the lens of the eye that blocks light from entering the eye.

Some people with MD may need surgery for scoliosis, or curvature of the spine. Certain medications can help delay damage to muscles or minimize the symptoms of MD.

These can include the following:. Some methods focus on correcting the function of a specific gene, while others rely on a genome-wide approach.



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