Current Treatments for Duchenne Muscular Dystrophy

Written by Margaret Anne Rockwood | Last updated June 5, 2026
Medically reviewed by Jonathan Soslow, MD

Over the past two decades, treatment of Duchenne Muscular Dystrophy (DMD) has shifted from purely supportive care to options that include pharmacologic and organ-specific therapies, as well as genetic interventions for some patients. Management of DMD focuses on slowing skeletal muscle decline, preserving respiratory function, and aggressively treating cardiomyopathy, which is the leading cause of death in DMD patients.

Corticosteroids for Slowing Progression

Corticosteroids remain a primary standard of care for DMD. Corticosteroids like prednisone and deflazacort are widely used to delay disease progression by targeting inflammation and unstable muscle cell membranes.

In DMD, the absence of dystrophin makes muscle cell membranes unstable, leading to repeated cycles of injury, inflammation, and incomplete repair. Corticosteroids dampen this chronic inflammatory response, which limits secondary muscle damage caused by immune cells and inflammatory signaling.

They also reduce fibrosis and fatty replacement of muscle tissue, helping preserve functional muscle fibers. In addition, corticosteroids may improve calcium handling and membrane stability within muscle cells, making them less susceptible to injury despite the lack of dystrophin. These combined effects slow the progression of muscle weakness and prolong muscle function.

The benefits of long-term corticosteroid use include:

• Delayed onset of respiratory decline and cardiomyopathy by several years, contributing to improved survival; modern cohorts treated with corticosteroids and comprehensive care often live into their late 20s or 30s, compared with the late teens without these treatments.
• ~ 2-3 years of prolonged ambulation, with some studies finding that some boys remain ambulatory into their mid-teens rather than losing the ability to walk around 10-12.
• Delay in loss of motor and cardiopulmonary function
• Delayed scoliosis

Outcomes vary depending on when treatment is started, the dosing regimen, and individual responses.

Managing the Downsides of Corticosteroids

While corticosteroids are indispensable because of these proven benefits, they carry significant side effects, including weight gain, growth suppression, bone fragility, and behavioral changes.

1. Optimizing Dosing Regimens

One of the most important strategies is adjusting how steroids are given:

• Daily vs. intermittent dosing: Daily dosing provides the strongest functional benefit. Intermittent regimens (e.g., 10 days on / 10 days off) can reduce side effects like weight gain and growth suppression but is inferior to daily dosing. High-dose weekend only steroids may be comparable to daily dosing, but long-term efficacy has not been evaluated.
• Using the lowest effective dose: Physicians may continually titrate dosing to balance function and tolerability. When using the lowest effective corticosteroid dose some reduction in efficacy is expected versus standard daily dosing.
• Switching agents: Deflazacort may cause less weight gain than prednisone, but deflazacort may have more pronounced effects on growth and the development of cataracts. Prednisone may be preferred in some cases for cost or availability.

2. Protecting Bone Health

Steroids significantly increase the risk of osteoporosis and fractures, due to several mechanistic changes, including impeding osteoblasts, increasing osteoclast activity, lowering calcium availability and lowering secretion of estrogen and testosterone. Care of DMD patients should include:

• Routine vitamin D and calcium supplementation
• Regular bone density monitoring (DEXA scans)
• Screening for fractures with lateral spine x-rays
• Early use of bisphosphonates if fractures or significant bone loss occur
• Encouraging weight-bearing activity as tolerated

3. Managing Weight Gain and Metabolic Effects

Weight gain is one of the most common and challenging side effects. Physicians can help their patients by:

• Referral to a dietician to help them achieve balanced nutrition with lower sugar intake and controlled portion sizes.
• Monitoring for insulin resistance or glucose intolerance and responding with combination of nutritional or corticosteroid adjustments and increased low-impact physical activity. Metformin or other metabolic interventions may be appropriate in mild-moderate cases, including insulin therapy, if hyperglycemia is significant.

4. Monitoring Growth and Puberty

Steroids can suppress growth and delay puberty, warranting:

• Regular height and growth tracking
• Endocrinology referral if growth failure is significant
• In some cases, growth hormone or testosterone therapy

5. Addressing Behavioral and Mood Changes

To address the irritability, mood swings, or attention issues that are frequently associated with corticosteroid use:

• Adjust dose timing (morning dosing reduces sleep disturbance)
• Behavioral therapy or school accommodations, if needed
• Medication adjustments if psychiatric effects are severe (rare)

6. Preventing and Monitoring Other Complications

Physicians should also screen for and manage systemic side effects, including:

• Cataracts, which are prevalent in DMD due to chronic steroids, calling for regular ophthalmology exams
• Hypertension, through routine blood pressure checks and medications if needed.
• Infection risk is higher, so vaccination schedules should be diligently followed.

• • Adrenal suppression, suspected by symptoms of fatigue, weakness disproportionate to progression, loss of appetite and weight loss, nausea or vomiting, abdominal pain or dizziness or orthostatic lightheadedness and headache. Because corticosteroids suppress the hypothalamic–pituitary–adrenal axis, gradual tapering (never abrupt stop) is essential. In addition, patients on chronic steroids are unable to mount an appropriate cortisol response, requiring stress dose steroids with minor or major stressors.

Steroid-Sparing Disease-Modifying Therapy

Alternative therapies are a strategy for managing symptoms as standalone therapy or in conjunction with a lowered corticosteroid dose. Paving the way is givinostat (Duvyzat), a histone deacetylase (HDAC) inhibitor, the first nonsteroidal agent approved in the U.S. for all genetic variants of DMD in patients aged 6 and older.

Givinostat modulates pathways involved in inflammation, fibrosis, and muscle regeneration, including downregulation of pro-inflammatory cytokine signaling (e.g., TNF-α, IL-6, NF-κB pathways), inhibition of fibro-adipogenic progenitor (FAP) activation and TGF-β–mediated fibrotic signaling, and promotion of muscle regeneration through enhanced satellite cell differentiation and myogenic gene expression (e.g., MyoD, myogenin).

In the phase 3 EPIDYS study, when added to background corticosteroid therapy, givinostat slowed functional decline by a mean of 1.78 seconds on 4-stair climb over 18 months (about a 59% relative reduction in worsening versus corticosteroids alone).

Valmorolone is an effective corticosteroid alternative for symptom management. While it is a steroid, because of its dissociative mechanisms, it offers anti-inflammatory benefits while avoiding the more severe side effects of steroids.

Respiratory and Multidisciplinary Support

As DMD progresses, respiratory muscle weakness leads to hypoventilation and increased risk of infection. Noninvasive ventilation, airway clearance techniques, and assisted coughing devices are standard components of care. These interventions have significantly reduced mortality from respiratory failure.

Multidisciplinary management—including physical therapy, orthopedic care, and nutritional support—remains essential. Advances in respiratory care have shifted the primary cause of death in DMD from pulmonary complications to cardiac disease.

Cardiac Therapies

Cardiomyopathy is now the leading cause of death in DMD and typically develops during adolescence.

In healthy individuals, dystrophin stabilizes the sarcolemma (cell membrane) in both skeletal and cardiac muscle. In DMD, the absence of dystrophin renders cardiomyocytes structurally fragile. Repeated mechanical stress from normal cardiac contraction and relaxation results in sarcolemmal disruption, calcium influx, myocyte necrosis and inflammation. Over time, the damaged myocardium is replaced by fibrosis and fatty infiltration, often beginning in the subepicardial region of the left ventricular free wall, ultimately leading to dilated cardiomyopathy.

Because this is the leading emergent threat in DMD, cardiac management has become a central pillar of treatment.

Standard Cardiac Medications

Current guidelines recommend early and proactive use of heart failure medications, even before symptoms appear. These include:

• Angiotensin converting enzyme inhibitors (ACEi) or angiotensin receptor blockers (ARB): Used prophylactically at 8 years of age or with any cardiac abnormalities on echocardiography or cardiac MRI
• Mineralocorticoid receptor antagonists (e.g., eplerenone, spironolactone): Help reduce myocardial fibrosis and preserve cardiac function, started with the detection of myocardial fibrosis, left ventricular dysfunction, or prophylactically around 10 years of age
• Beta-blockers: Recommended once systolic dysfunction develops or with persistent tachycardia
• Angiotensin receptor blocker/neprilysin inhibitor (ARNI): While no large studies evaluating ARNIs in DMD exist, providers have begun using ARNIs in DMD patients with significant LV dysfunction (usually LVEF≤40%) based on clinical trials in adults with cardiomyopathy.
• Sodium-glucose cotransporter 2 inhibitors (SGLT2i): SGLT2i’s have become the fourth pillar of guideline-directed medical therapy. Though not well-evaluated in DMD, SGLT2i’s are recommended in DMD patients with LVEF<40% and have begun to be prescribed in DMD patients with milder cardiomyopathy.

These therapies have improved outcomes by slowing progression of cardiomyopathy, with randomized data showing attenuation of decline in LV function when mineralocorticoid antagonists are added to ACEi/ARB therapy.

Studies demonstrate delayed onset of left ventricular dysfunction and attenuation of functional decline (e.g., ~50% reduction in decline in LV strain with eplerenone), contributing to significant survival gains. Optimal dosing and timing are still being refined.

Exon-Skipping Therapies

Exon-skipping therapies are now established treatments for specific genetic subtypes of DMD that have out-of-frame (reading frame) deletions.

These drugs use antisense oligonucleotides (ASOs) to modify pre-mRNA splicing, allowing cells to bypass mutated exons. This restores the reading frame of the dystrophin gene, turning the exons into in-frame transcripts. The resulting dystrophin protein is shorter but partially functional, resembling that seen in Becker muscular dystrophy.

A key limitation of exon-skipping therapies is that they are mutation-specific, so only subsets of patients benefit. Current exon-skipping therapies only address 25-30% of DMD patients. These include Eteplirsen (exon 51, ~13% of cases), Golodirsen / Viltolarsen (exon 53, ~8% of cases), and Casimersen (exon 45, ~8% of cases). Treatment requires ongoing administration and produces variable levels of dystrophin restoration.

The remaining ~70–75% of DMD patients include those with out-of-frame deletions as well as non-deletion mutations (duplications, nonsense mutations, and small sequence variants). ASO interventions for more exon subtypes are in trials.

While these therapies do not cure DMD, they restore small amounts of dystrophin in patients with certain mutations. The magnitude of clinical benefit is variable and still evolving.

AAV Gene Therapy

Gene therapy has become part of current clinical practice for selected patients in very recent years and represents a major step forward in treating the underlying cause of DMD.

Approved in 2023, delandistrogene moxeparvovec (Elevidys) uses an adeno-associated viral (AAV) vector to deliver a shortened microdystrophin gene into the bloodstream. This treatment is administered as a one-time infusion to enable long-term production of dystrophin in muscle cells.

Access is limited by specific eligibility criteria, including age, stage, and immune status (not having high levels of antibodies to the viral vector). Clinical studies have shown increased microdystrophin expression. While 1-year clinical data was underwhelming, recently released 2-year and 3-year data have demonstrated stabilization in multiple clinical endpoints that would not be expected in the natural history of disease. Long-term durability and safety remain areas of active monitoring.

An important consideration is that patients can currently only receive one infusion because antibodies to the AAV vector are expected to develop, preventing re-dosing. So, the dilemma is whether it is better to treat early, potentially benefitting skeletal and heart muscle sooner, or to wait for future iterations that may offer broader or more durable benefits.

Together, these current treatments have transformed DMD from a uniformly fatal childhood disorder into a condition where patients increasingly survive into adulthood, with improved quality of life and more time and reason for hope from what is a rapidly expanding landscape of therapies.

References

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