Muscular dystrophy refers a group of disorders that involve a progressive loss of muscle mass and consequent loss of strength. The main forms of muscular dystrophy may affect up to 1 in every 5,000 males. The most common form is Duchenne muscular dystrophy. It typically affects young boys, but other variations can happen in adulthood. Muscular dystrophy is caused by genetic mutations that interfere with the production of muscle proteins that are needed to build and maintain healthy muscles. There is currently no cure, but certain physical and medical treatments can improve symptoms and slow the progression.
Muscular dystrophy causes the gradual weakening of skeletal muscle. Here are the symptoms of Duchenne muscular dystrophy, the most common form of the disease. Early symptoms include:
- a waddling gait
- pain and stiffness in the muscles
- difficulty with running and jumping
- difficulty sitting up or standing
- learning disabilities, such as developing speech later than usual
Doctors use different types of steroids to treat different conditions. In Duchenne, a type of steroids called glucocorticosteroids is used to help maintain muscle strength. They are different from the anabolic steroids that are sometimes misused by athletes who want to become much stronger. Steroids are the only medicines currently available that can slow down the muscle damage and weakness caused by Duchenne.
- Prednisone (prednisolone) is the only steroid approved by the U.S. Federal Drug Administration (FDA) and available in the US. The starting dose of Prednisone is 0.75mg/kg/day.
- Deflazacort is a steroid available to treat Duchenne outside the US. The starting dose of deflazacort is 0.9 mg/kg/day.
- Albuterol is an immunosuppressant drug that’s widely utilized as an inhalant form for asthmatic.
- Creatine is another nutritional supplement that helps the patient’s body to build up the muscle’s energy supply.
- Anabolic steroids play a significant role in building the body tissues by compensating for muscle loss.
- Calcium blockers are common steroids used for treating the symptoms of muscle dystrophy.
Here is the recommended standard care for using steroids. Talk to your doctor about care options that go above standard care.
Know when to start taking steroids.
Your doctor should start giving your child steroids when he reaches the “plateau phase” or sooner. In Duchenne, the plateau phase is when your son:
- has learned all of his motor skills (crawling, walking, climbing stairs),
- is having minimal trouble walking,
- can rise from the floor with little to no effort, and
- can climb stairs with little to no effort.
In other words, when your child is still similar to his peers physically, that is the time to intervene with steroids. After this plateau stage, it will become more difficult for your child to walk, rise from the floor, and climb stairs. If your doctor does not offer steroids during your child’s plateau stage, your doctor should offer your child steroids when he starts to be less active.
The outlook will depend on the type of muscular dystrophy and how severe the symptoms are. Duchenne muscular dystrophy can lead to life-threatening complications, such as breathing difficulties and heart problems. In the past, people with this condition did not usually survive beyond their 20s, but progress is improving the outlook. Currently, the average life expectancy for people with Duchenne is 27 years, and it may improve in time, as treatment progresses.