Muscular Dystrophy Treatments: Current Options and Emerging Advancements

People with muscular dystrophy experience gradual loss of muscle strength, which can cause problems like difficulty walking. But muscular dystrophy can affect other parts of the body as well, such as the heart, bones, lungs, and digestive tract. 

There’s no cure for muscular dystrophy, but there are medications and treatments to help delay the progression of muscle weakness and manage some of the health issues the condition causes. 

What is muscular dystrophy?

Muscular dystrophy refers to a group of genetic diseases that cause progressive muscle weakness. Muscular dystrophy can be inherited, which means it’s passed through a family, or it may occur spontaneously, which means that no one else in the family has it.

People with muscular dystrophy may have symptoms like difficulty walking, frequent falls, and trouble getting up from a seated position. As the disease progresses, they may have difficulty breathing or have heart problems. 

There are many types of muscular dystrophy. The type of muscular dystrophy a person has will determine when and how they develop symptoms. The most common types of muscular dystrophy are: 

• Duchenne muscular dystrophy: This is one of the most severe forms of muscular dystrophy. Duchenne muscular dystrophy affects children, and symptoms usually begin before age 5. 

• Becker muscular dystrophy: It’s very similar to Duchenne muscular dystrophy, but symptoms are less severe and may not begin until adulthood. 

• Congenital muscular dystrophy: Symptoms can start at birth. Babies with congenital muscular dystrophy have low muscle tone or “floppiness.”

• Limb-girdle muscular dystrophy: Muscles in the back and shoulders are mostly affected. Symptoms can start in childhood or adulthood.

• Facioscapulohumeral muscular dystrophy: This condition causes muscle weakness of the face and shoulders that usually starts in young adulthood. 

• Myotonic dystrophy: This is the most common form of muscular dystrophy in adults. In addition to muscle weakness, patients with myotonic dystrophy may develop other health conditions like cataracts, diabetes, or thyroid dysfunction. 

• Oculopharyngeal muscular dystrophy: This condition leads to weakening of the muscles around the eyes and throat. It starts in adulthood. 

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How is muscular dystrophy treated? 

There’s no cure for muscular dystrophy. But there are treatments available to help improve muscle function and manage complications that develop due to muscle weakness. Most people with muscular dystrophy will use a combination of treatment options including:

• Medication

• Physical therapy

• Assistive devices 

Let’s go through some of the problems muscular dystrophy causes, and how they’re treated.  

Treatment for muscle weakness 

There’s no therapy that can completely prevent muscles from becoming weak in people with muscular dystrophy. But there are medications that can delay this process. Treatment options for people with muscular dystrophy include:

• Corticosteroids: These medications reduce inflammation and tamp down the immune system. These actions help slow the progression of muscle weakness. There are many types of corticosteroids used to treat muscular dystrophy including prednisone and deflazacort. 

• Gene therapy: Children with Duchenne muscular dystrophy have a genetic mutation that prevents them from making a protein called dystrophin. Dystrophin is important for maintaining muscle health. The goal of gene therapy is to introduce a healthy copy of the gene for dystrophin so that the protein can be produced. The FDA approved Elevidys as gene therapy for people with Duchenne muscular dystrophy.

• HDAC inhibitor: In 2024, the FDA approved Duvyzat (givinostat) to treat children 6 years and older with Duchenne muscular dystrophy. It’s not clear how Duvyzat lowers inflammation and slows down muscle loss. It’s given by mouth twice daily. 

• Exon-skipping therapy: Another group of medications, called exon-skipping therapy, can help people with Duchenne muscular dystrophy produce more dystrophin protein. Exon-skipping therapy works by encouraging the body to “skip over” the damaged part of the gene involved in muscular dystrophy. Exon-skipping therapies that have been approved to treat Duchenne muscular dystrophy include: Exondys 51, Vyondys 53, Viltepso, and Amondys 45.

Most people with muscular dystrophy benefit from other forms of treatment, including:

• Physical therapy: Working with a physical therapist can help people maintain muscle strength and flexibility. 

• Occupational therapy: Occupational therapists teach people with the condition how to perform physical tasks at home or work as their muscle strength changes. 

• Physical activity: Regular physical activity can also help delay muscle weakness. 

• Support aids: People with muscular dystrophy may maintain their mobility by using supportive aids like braces, splints, walkers, or wheelchairs. 

Breathing treatment

Muscular dystrophy can weaken breathing. When you breathe you use different muscles in your chest wall and your diaphragm to move air in and out of your lungs. People with muscular dystrophy can develop weakness in the muscles needed for breathing. Over time, the lungs can become more stiff and less able to fill with air. Muscle weakness can also make it harder for people to clear their airway by coughing. This means people with the conditions are more likely to develop infection of the lungs. 

Respiratory treatment for muscular dystrophy includes: 

• Respiratory therapy: Just like other muscles can be strengthened with physical therapy, people with muscular dystrophy may use respiratory therapy exercises to help maintain the muscles needed for breathing. Respiratory therapy also includes techniques to help keep the airways clear. 

• Antibiotics: People with muscular dystrophy may be at increased risk of developing lung infections. If an infection develops, a healthcare professional will prescribe antibiotics to treat the infection. 

• Noninvasive ventilation: As the muscles in the chest and the diaphragm weaken, people with muscular dystrophy may need additional support for breathing. Noninvasive ventilation uses pressure to help inflate the lungs, so that people can breathe more easily. Some options include continuous positive airway pressure (CPAP) and bilevel positive airway pressure (BiPAP) machines. 

Heart health

People with muscular dystrophy may also develop heart problems, such as:

• Cardiomyopathy or weakness of the heart muscle

• Heart failure when the heart is no longer able to pump enough blood to the rest of the body

• Arrhythmias or abnormal heart rhythms

Treatments for heart problems in people with muscular dystrophy include:

• ACE inhibitors: ACE inhibitors are a type of blood pressure medication that can also help to treat some heart diseases. Research shows that these medications help protect the hearts of people with Duchenne muscular dystrophy. 

• Implantable cardiac defibrillator: An implantable cardiac defibrillator, or ICD, is a device placed in the chest, beneath the skin, that monitors heart function and can shock the heart if it goes into an unstable rhythm. Some ICDs are also programmed to regulate the heartbeat and act as a pacemaker. 

Bone health

People with muscular dystrophy may develop problems with bone strength as a result of long-term treatment with corticosteroids. In order to maintain bone strength, people with muscular dystrophy may get the following:

• Calcium and vitamin D: Calcium is an important mineral for building healthy bones. And vitamin D helps our body to absorb calcium from the foods we eat. Medical professionals may prescribe supplemental calcium or vitamin D in order to make sure levels are high enough to support the bones. 

• Bisphosphonates: This is a group of medications used to treat osteoporosis or weak bones. These medications slow down bone loss. 

Digestive support 

The muscles around the intestine help move food through the digestive tract. People with muscular dystrophy may have difficulty with digestion, and can experience problems, such as:

• Difficulty swallowing

• Gastroesophageal reflux

• Constipation 

• Gastroparesis (slow movement of food through the stomach and small intestine)

Managing gastrointestinal problems caused by muscular dystrophy includes treatments like:

• Laxatives or enemas to manage constipation

• H2 receptor agonists or proton pump inhibitors (PPIs) to reduce gastric reflux symptoms 

• Dietary changes, such as eating smaller, more frequent meals

In severe cases, if people aren’t able to get enough food by eating, they may benefit from getting a feeding tube. 

Are there any new and upcoming treatments for muscular dystrophy?

Researchers are studying new treatments for muscular dystrophy. Once a new medication is developed, it’s studied in clinical trials to make sure it’s safe and effective. Treatments that are currently being studied in clinical trials include new gene therapies, steroids, and monoclonal antibodies. 

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Written by:

Christine Giordano, MD

Christine Giordano, MD, is board-certified in general internal medicine. She received her medical degree from Rutgers New Jersey Medical School and completed residency at Thomas Jefferson University.

Edited by:

Patricia Pinto-Garcia, MD, MPH

Patricia Pinto-Garcia, MD, MPH, is a medical editor at GoodRx. She is a licensed, board-certified pediatrician with more than a decade of experience in academic medicine.

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