When your child has Duchenne muscular dystrophy, there’s no way to cure it. But there are medications to help with different parts of the disease. Because of these medicines, people with DMD are living longer and better than ever before.
Steroids (Corticosteroids)
People with DMD will likely take steroids starting at a young age. Steroids help make it so the condition worsens more slowly. It’s usually best to start them before the physical symptoms of DMD get serious. Some steroids are specially approved to treat DMD.
Studies have shown that steroids can improve strength. They also may help muscles work better. As a result, you can do things like climb stairs faster. Lung function may improve, too.
Steroids also make it less likely that your child's spine will curve (scoliosis). They may make you more able to walk for longer. Some studies suggest people with DMD also live longer when they take steroids. But not all studies back this up. Ask your child's doctor which steroid they recommend and why.
Exon-Skipping Drugs
People with DMD have a change (mutation) in the gene for a protein called dystrophin, which helps protect muscles as you move. The mutations stop your body from making the protein, which leads to muscle damage.
This is where exon-skipping drugs come in. Exons are sections of a gene, and some on your dystrophin gene are missing when you have DMD. Exon-skipping drugs are made of tiny snippets of genetic material that help your body "skip" over missing exons when it makes dystrophin. This allows your cells to make a protein that's shorter than it should be, but that still works at least somewhat.
Exon-skipping drugs could potentially help lots of people with DMD. But people with DMD need drugs that skip different parts of the gene, depending on where their mutation is. The FDA has approved drugs that skip exons 45, 51, and 53:
- Casimersen (Amondys 45)
- Eteplirsen (Exondys 51)
- Golodirsen (Vyondys 53)
- Viltolarsen (Viltepso)
Keep in mind, though, that these drugs won’t work for everyone. About 8% of people with DMD have a mutation that means it may help to skip exon 45. About 13% could benefit from skipping exon 51. Another 8% have changes that mean it could help to skip exon 53.
Your child will need genetic testing to find out which mutation they have and whether an exon-skipping drug can help. The test results also will tell you whether they may be able to take part in a clinical trial to test a drug that isn’t approved yet.
Heart Medicines
When your child has DMD, their doctor will check their heart regularly to see how well it’s working. They might need to take heart medicines to help with your blood pressure and heart function. Heart medicines that may be prescribed for people with DMD include:
- Angiotensin-converting enzyme (ACE) inhibitors
- Angiotensin receptor blockers
- Beta-blockers
- Aldosterone antagonists
Gastrointestinal Medicines
Many people with DMD have chronic constipation. Your doctor may suggest stool softeners to help with this.
People who take steroids for DMD may end up with acid reflux or heartburn. Antacids or proton pump inhibitors can help with this side effect.
Bone-Strengthening Medicines
DMD can lead to weakened bones that break more easily than they should. Your child's doctor may recommend medicines called bisphosphonates or other types of drugs to strengthen their bones and treat osteoporosis.
Gene Therapy
Researchers hope that one day, gene therapy can help with many diseases that are caused by single genes. Duchenne muscular dystrophy is no exception. The idea is to correct the genetic mutation that causes the condition.
The FDA has agreed to a sped-up approval review process for one such treatment for DMD, called SRP-9001. Clinical trials are underway for this therapy, which could be used for people with DMD who are able to walk.
What About Clinical Trials?
Clinical trials can be a way to expand the treatment options for DMD. Sometimes a trial may help improve health and quality of life. But there are no guarantees.
If you’re interested in clinical studies or trials, ask your doctor what’s available and if your child might be a good candidate for one. You can also search for clinical trials in DMD on the U.S. National Library of Medicine's clinicaltrials.gov website.
DMD Care Team
Because DMD affects so many areas of life, it's important to have a team of health care providers to make the most of your child's health. It's a good idea to have a care coordinator to act as a point person. They'll need a neuromuscular specialist as well as a primary doctor and dentist. A cardiologist can help with heart problems related to DMD. A pulmonologist can help with breathing and lung issues.
Physical and occupational therapists help them stay as mobile and active as possible. Children with DMD who have learning disorders may be referred to a neuropsychologist. Depending on your child's needs, their team may also include others, such as a developmental pediatrician, endocrinologist, and/or gastroenterologist.
Show Sources
Photo Credit: Jose Luis Pelaez Inc / Getty Images
SOURCES:
Cleveland Clinic: “Duchenne muscular dystrophy.”
Muscular Dystrophy Association: “Duchenne Muscular Dystrophy (DMD).”
Parent Project Muscular Dystrophy: “Mutation-Specific Therapies,” “Medications & Supplements (Other Than Steroids,” "Assembling a Care Team," "SRP-9001."
FDA: “FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation.”
Frontiers in Bioengineering and Biotechnology: “A Decade of Progress in Gene Targeted Therapeutic Strategies in Duchenne Muscular Dystrophy: A Systematic Review.”
Clinicaltrials.gov: “Affinity Duchenne: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD).”
University of Rochester Medical Center: “Experimental Gene Therapy Targets Duchenne Muscular Dystrophy.”
News release, Sarepta Therapeutics, Nov. 28, 2022.
