Muscular Dystrophy
Treatment

Medications

Currently, Duchenne MD is the only form of MD with research evidence showing that medication can be beneficial. Steroids — also called glucocorticoids or corticosteroids — are the only drugs known to slow the decline in muscle strength and motor function in DMD. The goal of steroid use is to help the child walk independently for longer, to allow more participation in everyday activities, and to later minimize breathing, heart and orthopedic problems. They can also reduce the risk of scoliosis, an abnormal curvature of the spine.

Steroids do have side effects, including weight gain, behavioral issues, bleeding in the gastrointestinal tract, high blood pressure and the development of diabetes. Children need to be followed closely when on this medication and cannot stop taking it suddenly.

Albuterol has also been tested in DMD as well as other neuromuscular disorders, and evidence suggests some mild benefit in increasing lean muscle mass. Some patients also feel an increase in energy. Albuterol has few side effects, most commonly anxiety, agitation and tremor.

Various supplements have also been tested and may be helpful for some MDs. These include antioxidants, such as coenzyme Q10, and creatine monohydrate.

Physical Therapy

People with MD need different types of rehabilitation management throughout their lives. Much of this care will be delivered by physiotherapists and occupational therapists, but other experts may be involved, including rehabilitation specialists, orthotists, providers of wheelchairs and orthopedic surgeons. Management of muscle extensibility and joint contractures is a key part of rehabilitation management.

The goal of stretching is to preserve function and maintain comfort. The stretching program will be monitored by the physiotherapist but needs to become part of the family's daily routine. There are many factors that contribute to the tendency for joints to get tight or "contracted." These include the muscle becoming less elastic due to limited use and positioning, or the muscles around a joint becoming out of balance, with one stronger than another. Maintaining good range of movement and symmetry at different joints is important. This helps to maintain the best possible function, prevent the development of fixed deformities, and prevent pressure problems with the skin.

As difficulty with walking increases, a power wheelchair should be provided sooner rather than later. With time, arm strength becomes more of an issue. Physiotherapists and occupational therapists can recommend assistive devices to help maintain independence.

It is important to think proactively about the kind of equipment that will best support independence and participation in everyday activities, and to plan ahead to provide it in as timely a manner as possible. Additional adaptations may be needed to help patients with getting upstairs and transferring from one place to another (for instance, from the bed to a wheelchair), eating and drinking, turning in bed and bathing.

Bone health is also very important, especially for children who don't stand much during the day and those taking steroids. These children have a lower bone mineral density and are at increased risk of fractures (broken bones) compared to the general population. Sometimes vitamin D and calcium are prescribed.

Pulmonary Care

Because breathing muscles can become affected as patients with MD get older, they are at risk for chest infections, often due to an ineffective cough. Later on they can develop problems with their breathing when sleeping. Some may require help with breathing during the day as well. Immunizations are recommended to decrease the chance of developing significant problems from viruses such as influenza.

As this is a staged progression of problems, a planned and proactive approach to respiratory care is possible based on appropriate surveillance, prevention and interventions. This is why the patient care team at the UCSF Pediatric Neuromuscular Clinic includes a pulmonologist and respiratory therapist with expertise in non-invasive ventilation and associated techniques for increasing the amount of air that can enter the lungs, and manual and mechanically assisted cough.

Cardiac Care

Some MDs are associated with heart problems such as dilation, leading to heart failure or electrical conduction issues. The aim of cardiac management in MD is early detection and treatment of the deterioration of heart muscle function — usually cardiomyopathy — that often occurs as some of the diseases progress.

As these problems often develop silently, without any significant symptoms, they must be watched for carefully so they can be treated promptly. Surveillance and proactive management are key. For this reason, the UCSF Pediatric Neuromuscular Clinic's patient care team includes a cardiologist.

Nutrition and Gastrointestinal Care

Nutrition and weight are key issues in keeping people with MD healthy. Some patients will develop swallowing problems. At UCSF, we work closely with a number of gastroenterologists and nutritionists to help manage these problems.

Reviewed by health care specialists at UCSF Benioff Children's Hospital.

Related Information

UCSF Clinics & Centers

Pediatric Brain Center

Neuromuscular Clinic
1825 Fourth St., Sixth Floor
San Francisco, CA 94158
Phone: (415) 353-7596
Fax: (415) 353-2400
Appointment information

Patient Experiences