Duchenne Muscular Dystrophy Market

Market Overview:

The 7 major Duchenne muscular dystrophy markets reached a value of USD 2,259.0 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 3,906.1 Million by ​2035​, exhibiting a growth rate (CAGR) of 5.11% during 2025-2035.

The Duchenne muscular dystrophy market has been comprehensively analyzed in IMARC's new report titled "Duchenne Muscular Dystrophy Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Duchenne muscular dystrophy (DMD) is a disorder that weakens skeletal and cardiac muscle and worsens over time. It is the most frequent type of muscular dystrophy. This ailment primarily affects boys, but girls who are carriers of DMD may experience milder symptoms. Indications of muscle weakness typically begin between the ages of 2 and 4 years but can occur as late as 6 years. Individuals suffering from this condition might experience problems, such as difficulty walking or climbing stairs, frequent falling, waddling or walking on toes, large calves as a toddler, weakness in the shoulders and pelvis, clumsiness, etc. The disorder can also cause cognitive impairment and learning differences, developmental delay, and scoliosis (spine curvature). Diagnosing DMD commonly involves a comprehensive blood test, along with a physical and neurological exam. The healthcare provider may utilize a genetic test that looks for a complete or near-complete absence of the dystrophin gene to confirm the diagnosis of the condition. Furthermore, muscle biopsy may be recommended to visually examine muscle tissue for characteristic changes seen in DMD.

The escalating prevalence of the mutation in the gene, which prevents the body from producing enough of the protein dystrophin that helps protect muscle fibers, is primarily driving the Duchenne muscular dystrophy market. In addition to this, the inflating utilization of corticosteroids, such as prednisolone and prednisone, to reduce inflammation and delay the onset of cardiomyopathy and respiratory insufficiency in patients is also creating a positive outlook for the market. Moreover, the widespread adoption of physical therapy that includes regular stretching exercises to maintain joint mobility and prevent contractures is further bolstering the market growth. Apart from this, the rising usage of assistive devices, like ankle-foot orthoses (AFOs), which maintain proper foot position and support the ankle and foot, thereby helping people with weakened muscles to walk more easily, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of gene therapy, since it aims to deliver a functional, albeit shortened version of the dystrophin protein (micro-dystrophin) to muscle cells using a viral vector to restore muscle function, is expected to drive the Duchenne muscular dystrophy market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the Duchenne muscular dystrophy market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for Duchenne muscular dystrophy and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Duchenne muscular dystrophy market in any manner.

Recent Developments:

• In January 2025, Sarepta Therapeutics reported positive topline results from Part 2 of EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled Phase 3 clinical trial of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy.
• In November 2024, REGENXBIO Inc. reported that AFFINITY DUCHENNE, a multi-center, open-label trial of RGX-202, a possible best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne), had reached the pivotal stage and dosed the first patient. The company also released new, promising efficacy and safety results from the Phase I/II section of the study, which included the first functional data.
• In October 2024, PTC Therapeutics reported that the U.S. Food and Drug Administration (FDA) had approved for evaluation the resubmission of the New Drug Application (NDA) for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy.
• In March 2024, the U.S. FDA approved Duvyzat (givinostat) oral medication for the treatment of Duchenne muscular dystrophy in patients six years of age and older. Duvyzat is the first nonsteroidal medication approved to treat patients with all genetic variants of DMD.

Drugs:

Duvyzat (givinostat) is a histone deacetylase (HDAC) inhibitor used to treat Duchenne muscular dystrophy in people aged six and up. DUVYZAT inhibits enzymes known as histone deacetylases, which are involved in turning genes on and off within cells and can slow muscle regeneration in Duchenne. The drug may aid in activating muscle repair pathways by reducing HDAC activity, resulting in increased muscle fiber regeneration, less inflammation, and diminished fibrosis.

Elevidys (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy. The drug works by delivering gene therapy to muscle cells using a harmless AAV vector, instructing the cells to create a shorter variant of the dystrophin protein known as micro-dystrophin. This effectively addresses the absence of functional dystrophin in patients with DMD by supplying a partially functional protein to promote muscle function.

Emflaza (deflazacort) is the first glucocorticoid medication approved in the United States for the treatment of patients with Duchenne muscular dystrophy aged two years and older. Emflaza exerts its therapeutic effect by reducing inflammation and suppressing the immune system, thereby slowing the progression of muscle weakness and functional decline in patients by acting through the glucocorticoid receptor once metabolized into its active form. The medicine prevents the loss of muscular strength and function while also maintaining heart and respiratory function. Additionally, it lowers the occurrence of spinal curvature in persons with DMD.

PTC124 (Ataluren) is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. The drug allows ribosomal readthrough of mRNA with premature stop codons, which would otherwise cause protein chains to prematurely terminate. The use of ataluren allows cellular machinery to bypass nonsense mutations in genetic material, continue translation, and therefore restore the synthesis of a full-length functioning protein.

RGX-202 is a gene therapy that allows muscle cells to produce a functioning, shorter version of the dystrophin protein (known as microdystrophin). This protects muscle fibers from damage, reducing the consequences of Duchenne muscular dystrophy by addressing the disease's underlying genetic basis. This is accomplished with a single administration of an AAV vector that is particularly targeted to muscle tissue.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the Duchenne muscular dystrophy market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the Duchenne muscular dystrophy market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current Duchenne muscular dystrophy marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the Duchenne muscular dystrophy market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the Duchenne muscular dystrophy market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the Duchenne muscular dystrophy market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (​2019-2035​) of Duchenne muscular dystrophy across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of Duchenne muscular dystrophy by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of Duchenne muscular dystrophy by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with Duchenne muscular dystrophy across the seven major markets?
• What is the size of the Duchenne muscular dystrophy patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of Duchenne muscular dystrophy?
• What will be the growth rate of patients across the seven major markets?

Duchenne Muscular Dystrophy: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for Duchenne muscular dystrophy drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Duchenne muscular dystrophy market?
• What are the key regulatory events related to the Duchenne muscular dystrophy market?
• What is the structure of clinical trial landscape by status related to the Duchenne muscular dystrophy market?
• What is the structure of clinical trial landscape by phase related to the Duchenne muscular dystrophy market?
• What is the structure of clinical trial landscape by route of administration related to the Duchenne muscular dystrophy market?