Quick Links
About myMDteam
Powered By
Muscular dystrophy (MD) treatments vary depending on the specific type of muscular dystrophy and its associated symptoms. Thanks to advancements in research, people with MD are experiencing better quality of life. New medications target specific gene mutations (alterations in DNA) that cause muscular dystrophy. Supportive treatments like braces and mobility devices empower individuals to maintain independence and mobility.
This article will cover nine treatment options for those living with muscular dystrophy. The most suitable treatment for you or your loved one may be different from others. Consulting with a neurologist who specializes in neuromuscular diseases can help.
Medications for muscular dystrophy are a recent development. New therapies focus on treating the disease at its source. Other drugs focus on reducing inflammation that contributes to muscle damage and weakness.
Dystrophin is a critical protein needed for muscle contractions. This long and specialized molecule connects the center of muscle cells to the edges. Without enough dystrophin, muscle cells can’t hold together and work properly.
People with Duchenne muscular dystrophy (DMD) can’t produce dystrophin, due to genetic changes in the dystrophin gene. This results in severe muscle weakness.
Becker muscular dystrophy (BMD) is a milder form of muscular dystrophy. BMD develops in people who make dystrophin proteins that partially work.
Gene therapies work by targeting the abnormal DMD gene. The U.S. Food and Drug Administration (FDA) approved delandistrogene moxeparvovec (Elevidys) in 2023. It’s the first gene therapy for DMD, indicated for people 4 and up. This medication delivers genetic instructions for producing a shortened, functional version of the dystrophin protein. The body’s muscle cells use these instructions to make healthy proteins.
Researchers are still studying whether delandistrogene improves long-term mobility and muscle strength. The therapy is administered as a one-time intravenous (IV) injection. Common side effects include:
DMD and BMD develop in people with mutations affecting the DMD gene. Specifically, these people are missing exons, which are important pieces of genes that provide instructions to make proteins. You can think of exons like puzzle pieces. If you’re missing a piece, your puzzle won’t be complete. The same applies to missing parts of the DMD gene. Without all the exons, you can’t make healthy dystrophin proteins.
Exon-skipping therapies work by encouraging cells to “skip over” the missing exon during protein production. These drugs contain tiny proteins that patch over the hole. This tricks the cell into making healthy dystrophin proteins.
This approach is tailored to the specific exon that is missing. Doctors use genetic testing to diagnose DMD and BMD. These tests help find which exon is missing so you can get the correct treatment. Commonly skipped exons include 44, 45, 51, and 53.
The FDA has approved four exon-skipping therapies for treating muscular dystrophy. They include:
Common side effects of exon-skipping therapies include:
Without enough dystrophin protein, the skeletal muscles start breaking down over time. The immune system overreacts to the damaged muscle tissue and creates inflammation. Chronic (long-term) inflammation creates even more damage, worsening muscular dystrophy symptoms.
Health experts have known for years that steroids can help slow down disease progression. Studies show that these drugs improve muscle strength and function. They also help prevent scoliosis (abnormal curvature of the spine).
Commonly prescribed steroids for DMD include:
While these medications work well, they can come with unwanted side effects. Examples include:
Nonsteroidal drugs provide an alternative to steroids for managing inflammation, offering fewer side effects while addressing muscle loss. The FDA approved givinostat (Duvyzat) in March 2024 as the first nonsteroidal drug for DMD.
Givinostat belongs to a class of medications known as histone deacetylase (HDAC) inhibitors. They work by blocking HDAC enzymes, proteins that regulate gene activity. By inhibiting these enzymes, the drug helps control inflammation and preserves muscle tissue. The drug is indicated for people ages 6 and up.
Studies show that the most common side effects of givinostat are:
Muscular dystrophy treatment also includes a variety of supportive therapies. These treatments don’t directly address muscular dystrophy. Instead, they help relieve symptoms and prevent complications.
Children and adults with muscular dystrophy can develop contractures. This refers to the shortening of the tendons and muscles supporting joints. Contractures are painful and make movement difficult.
Doctors and therapists usually recommend bracing to stretch and flex muscles. Braces also provide extra support to weak muscles. Ankle-foot orthoses keep the foot aligned while sleeping. This keeps tendons stretched to avoid contractures.
Standing is an important part of building and keeping muscle strength. Doctors and therapists recommend standing frames, walkers, and wheelchairs. Using a standing device can also improve bone health and circulation.
As muscular dystrophy progresses, movement may become more difficult. Many people with this disease use mobility aids to help them get around. Examples include walkers, canes, and wheelchairs.
People with muscular dystrophy are at risk of developing scoliosis as well. Braces offer added support and stability for curved spines. Custom braces or supports can be built into wheelchairs for comfort.
Muscular dystrophy causes progressive muscle weakness, meaning symptoms worsen over time. Physical therapists focus on building muscle strength through exercises. They also provide stretches to improve joint mobility and prevent contractures.
Occupational therapists help people with activities of daily living. This is important for maintaining you or your child’s independence with muscular dystrophy. Occupational therapy can help with:
In addition to causing muscle weakness, muscular dystrophy can negatively affect the lungs and heart. Health experts recommend children with muscular dystrophy see a cardiologist (heart specialist) regularly. DMD and BMD are associated with cardiomyopathy and heart failure. This is because dystrophin is an important protein in the heart muscle.
Myotonic dystrophy, congenital muscular dystrophy, and Emery–Dreifuss muscular dystrophy are also associated with heart problems. People with these diseases can develop:
Doctors may recommend medications to help treat heart problems. ACE inhibitors and beta-blockers help take pressure off the heart muscle and slow down heart rates. These medications work best for cardiomyopathy from DMD and BMD.
For those with abnormal heart rhythms, pacemakers can help. These devices give impulses to return the heart back into a regular rhythm. Antiarrhythmic drugs and beta-blockers also work well.
Muscular dystrophy weakens the breathing muscles over time. This makes it harder to keep the lungs clear of mucus and other secretions. Doctors recommend cough assist devices to get rid of any unwanted phlegm or secretions. Proper respiratory care is vital to maintain lung function and prevent complications like infections or difficulty breathing.
Some people may need ventilation devices to breathe better. Noninvasive devices include continuous positive airway pressure (CPAP) and bilevel positive airway pressure (BiPAP) devices. They use air pressure to help you breathe in and out. CPAP and BiPAP devices usually use masks or nasal pieces to deliver air pressure. Most people wear them at night while sleeping.
Invasive ventilation uses medical devices placed with surgery. They’re used when people need around-the-clock help to breathe properly. One example is a tracheostomy tube. This tube is placed through the trachea (windpipe) in the neck. It’s attached to a special machine that helps you breathe.
Spine-straightening surgery can be an effective treatment for scoliosis, according to the Muscular Dystrophy Association. They can help straighten the spine and prevent further curvature, especially in children.
Growing rods are special metal rods placed along the spine in young children. The rod holds the spine in place and straightens any curving.
The rods are adjusted over time as the child grows. Once the child stops growing, the rods are removed. The doctor then fuses (connects) the vertebrae (bones in the spine) together. This keeps the spine straight and prevents further curving.
Muscular dystrophy treatment has come a long way, offering new hope for individuals and families living with this condition. From groundbreaking medical therapies like gene therapy and exon-skipping medications to essential supportive interventions such as physical therapy, mobility aids, and heart care, each approach plays a critical role in managing symptoms and improving quality of life.
Because muscular dystrophy affects everyone differently, creating a personalized treatment plan is essential. Speak with a neurologist or care team specializing in muscular dystrophy to explore the most effective therapies and strategies for you or your loved one. By staying informed and working with health care experts, you can take proactive steps toward achieving better health and maintaining independence for as long as possible.
On myMDteam — the social network for people living with muscular dystrophy and their loved ones — members share their experiences, ask questions, and find support from others who understand the challenges of living with MD.
How has treatment impacted your quality of life or that of your loved one? What supportive therapies or devices have made the biggest difference in your day-to-day activities? Share your experiences in the comments below, or start a conversation by posting on your Activities page.
Sign up for free!
Continue with Facebook
Sign up with your email
Become a member to get even more
A myMDteam Member
A couple years back I was diagnosed with that rapid heartbeat