Duchenne Muscular Dystrophy Market to Witness Significant Expansion During the Forecast Period (2025–2034) Amidst Advances in Gene Therapy and Novel Drug Approvals

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New York, USA, July 28, 2025 (GLOBE NEWSWIRE) — Duchenne Muscular Dystrophy Market to Witness Significant Expansion During the Forecast Period (2025–2034) Amidst Advances in Gene Therapy and Novel Drug Approvals | DelveInsight
The Duchenne muscular dystrophy market is witnessing steady growth driven by advancements in gene therapies, exon-skipping drugs, and corticosteroid alternatives. Increasing diagnosis rates, rising awareness, and supportive regulatory pathways have accelerated therapy approvals. Additionally, key players continue to expand their pipelines, with several novel therapies in late-stage clinical trials.
DelveInsight's Duchenne Muscular Dystrophy Market Insights report includes a comprehensive understanding of current treatment practices, emerging Duchenne muscular dystrophy drugs, market share of individual therapies, and current and forecasted Duchenne muscular dystrophy market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].
Key Takeaways from the Duchenne Muscular Dystrophy Market Report According to DelveInsight's analysis, the total Duchenne muscular dystrophy market size is expected to grow positively by 2034.
The United States accounts for the largest market size of Duchenne Muscular Dystrophy, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan.
In 2024, the United States had the highest prevalence of DMD among the 7MM countries, with approximately 17,000 cases. This number is expected to grow at a steady CAGR.
cases. This number is expected to grow at a steady CAGR. Prominent companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others, are actively working on innovative Duchenne muscular dystrophy drugs.
and others, are actively working on innovative Duchenne muscular dystrophy drugs. Some of the key Duchenne muscular dystrophy therapies in the pipeline include NS-089/NCNP-02 (brogidirsen), TAS-205 (pizuglanstat), RGX-202, and others. These novel Duchenne muscular dystrophy therapies are anticipated to enter the Duchenne muscular dystrophy market in the forecast period and are expected to change the market.
Discover which Duchenne muscular dystrophy medications are expected to grab the market share @ Duchenne Muscular Dystrophy Market Report
Duchenne Muscular Dystrophy Market Dynamics
The Duchenne muscular dystrophy market dynamics are anticipated to change in the coming years. Glucocorticosteroids remain the mainstay treatment option for Duchenne muscular dystrophy, despite their associated adverse side effects, while newer corticosteroids like EMFLAZA face cost-effectiveness challenges in the United States; moreover, there is currently no cure or disease-reversing therapy available for non-ambulant DMD patients. However, ongoing research in gene therapy and genetic technologies offers promising opportunities for developing targeted and personalized treatments, alongside a wider commercial potential for therapies such as Capricor's CAP-1002 and Antisense's ATL1102, which are specifically aimed at improving upper limb function in DMD patients and addressing a broader patient population.
Furthermore, many potential therapies are being investigated for the treatment of Duchenne muscular dystrophy, and it is safe to predict that the treatment space will significantly impact the Duchenne muscular dystrophy market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate is expected to drive the growth of the Duchenne muscular dystrophy market in the 7MM.
However, several factors may impede the growth of the Duchenne muscular dystrophy market. The diagnosis of Duchenne muscular dystrophy is often complicated due to inadequate patient history, the use of inappropriate medical tests, and the presence of multiple comorbidities, frequently leading to delayed or incorrect diagnosis; additionally, the high cost of current and emerging therapies may limit patient access globally, while the rarity of DMD and its occurrence in the paediatric population contribute to a low availability of participants for clinical trials, posing further challenges to therapeutic development.
Moreover, Duchenne muscular dystrophy treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Duchenne muscular dystrophy market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Duchenne muscular dystrophy market growth.
Duchenne Muscular Dystrophy Treatment Market
The primary treatment approaches for Duchenne muscular dystrophy focus on restoring dystrophin production through gene replacement or mutation-targeted genetic therapies, stabilizing muscle membranes, enhancing compensatory protein expression, reducing inflammation, and promoting muscle regeneration.
In the United States, several therapies have received approval for DMD management, including AGAMREE (vamorolone), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), VILTEPSO (viltolarsen), and ELEVIDYS (delandistrogene moxeparvovec), among others.
DUVYZAT (givinostat), a histone deacetylase (HDAC) inhibitor, has been authorized for use in DMD patients aged six and older. Notably, it is the first FDA-approved non-steroidal drug applicable to all genetic forms of DMD. Givinostat works by inhibiting HDAC enzymes, which regulate gene expression and contribute to muscle inflammation and damage. By blocking these enzymes, givinostat seeks to reduce inflammation, support muscle repair, and slow muscle degeneration in DMD patients.
AMONDYS 45, or casimersen, developed by Sarepta Therapeutics, is an antisense oligonucleotide designed for patients with a specific DMD gene mutation suitable for exon 45 skipping. Casimersen employs exon-skipping technology, targeting the dystrophin gene pre-mRNA to exclude exon 45 during processing. This allows the creation of a shortened but functional dystrophin protein, which helps maintain muscle fiber structure and function, aiming to restore the disrupted reading frame and partially compensate for the dystrophin deficiency.
AGAMREE is a prescription therapy for children aged two and older with DMD. It is a novel anti-inflammatory drug that selectively binds the glucocorticoid receptor but modulates downstream effects, bypassing the 11β-HSD enzyme pathway associated with steroid-induced toxicity. This distinct mechanism potentially provides the anti-inflammatory benefits of corticosteroids while minimizing their harmful side effects, making AGAMREE a safer treatment option for pediatric and adolescent patients. AGAMREE was granted Orphan Drug designation in August 2014.
Learn more about the Duchenne muscular dystrophy treatment options @ Duchenne Muscular Dystrophy Treatment Guidelines
Duchenne Muscular Dystrophy Emerging Drugs and Companies
The Expected launch of potential therapies may increase the market size in the coming years, assisted by an increase in the diagnosed prevalent population of DMD. Owing to the positive outcomes of several products during the developmental stage by key players such as NS Pharma/Nippon Shinyaku (NS-089/NCNP-02), Taiho Pharma (TAS-205), REGENXBIO (RGX-202), and others.
NS-089/NCNP-02 is an antisense oligonucleotide jointly developed by Nippon Shinyaku and the National Center of Neurology and Psychiatry (NCNP). It is being explored as a treatment option for Duchenne muscular dystrophy (DMD) patients whose dystrophin gene mutations can be addressed through exon 44 skipping. In January 2025, NS Pharma announced that NCNP published the outcomes of a first-in-human, investigator-led study of NS-089/NCNP-02 (brogidirsen) in Cell Reports Medicine. This therapy is currently being investigated in a global Phase II clinical trial (NCT05996003) for DMD.
TAS-205 (pizuglanstat), discovered by Taiho Pharmaceutical, is a selective inhibitor of hematopoietic prostaglandin D synthase (HPGDS). It is under development for DMD treatment and works independently of the type of dystrophin gene mutation. By inhibiting HPGDS, which contributes to inflammation in muscle tissue, TAS-205 aims to slow the progression of motor decline in DMD patients. This drug is currently undergoing Phase III trials.
RGX-202 is a gene therapy designed to deliver a transgene encoding a novel microdystrophin that retains critical functional components of the C-terminal domain present in natural dystrophin. RGX-202 is in a Phase II/III pivotal trial. The therapy has been granted Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation by the FDA.
In November 2024, REGENXBIO announced that the AFFINITY DUCHENNE trial of RGX-202 has progressed to its pivotal stage, with the first patient receiving the treatment. The company shared positive safety and efficacy results from its earlier Phase I/II study, which included functional outcomes. REGENXBIO has also reached an agreement with the FDA on the pivotal study design and the potential for accelerated approval, with a Biologics License Application (BLA) filing targeted for 2026. The pivotal trial is currently enrolling ambulatory patients aged 1 year and older.
The anticipated launch of these emerging Duchenne muscular dystrophy therapies are poised to transform the Duchenne muscular dystrophy market landscape in the coming years. As these cutting-edge Duchenne muscular dystrophy therapies continue to mature and gain regulatory approval, they are expected to reshape the Duchenne muscular dystrophy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.
To know more about new treatment for Duchenne muscular dystrophy, visit @ Duchenne Muscular Dystrophy Management
Recent Developments in the Duchenne Muscular Dystrophy Market In May 2025, Capricor Therapeutics completed a mid-cycle review meeting with the FDA for its Biologics License Application (BLA) for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA confirmed no significant deficiencies in the review and affirmed that the application is on track for a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. An advisory committee meeting is also planned, but no date has been set yet.
completed a mid-cycle review meeting with the FDA for its Biologics License Application (BLA) for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA confirmed no significant deficiencies in the review and affirmed that the application is on track for a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. An advisory committee meeting is also planned, but no date has been set yet. In March 2025, the FDA accepted Capricor Therapeutics' application for approval of deramiocel, a cell therapy developed to treat heart muscle disease in individuals with Duchenne muscular dystrophy (DMD), and granted it priority review to accelerate the agency's decision.
the FDA accepted Capricor Therapeutics' application for approval of deramiocel, a cell therapy developed to treat heart muscle disease in individuals with Duchenne muscular dystrophy (DMD), and granted it priority review to accelerate the agency's decision. In January 2025, Capricor Therapeutics announced the submission of its Biologics License Application (BLA) to the FDA for full approval of deramiocel, an investigational cell therapy for treating Duchenne muscular dystrophy cardiomyopathy.
announced the submission of its Biologics License Application (BLA) to the FDA for full approval of deramiocel, an investigational cell therapy for treating Duchenne muscular dystrophy cardiomyopathy. In November 2024, Regenxbio is advancing its gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) into pivotal development, with plans for a BLA submission by 2026. The company has secured FDA alignment on an accelerated approval pathway, proposing RGX-202 as a one-time treatment, potentially challenging Sarepta's Elevidys in the DMD market.
is advancing its gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) into pivotal development, with plans for a BLA submission by 2026. The company has secured FDA alignment on an accelerated approval pathway, proposing RGX-202 as a one-time treatment, potentially challenging Sarepta's Elevidys in the DMD market. In November 2024, Cumberland Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation and Rare Pediatric Disease Designation to Ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
Duchenne Muscular Dystrophy Overview
Duchenne muscular dystrophy (DMD) is a severe, progressive genetic disorder characterized by muscle degeneration and weakness. It primarily affects boys, with symptoms typically becoming evident between the ages of 2 and 5. DMD is caused by mutations in the DMD gene located on the X chromosome, which encodes for dystrophin, a crucial protein that helps stabilize and protect muscle fibers during contraction. The absence or dysfunction of dystrophin leads to muscle damage, inflammation, and eventual replacement of muscle tissue with fat and fibrotic tissue.
Clinically, children with Duchenne Muscular Dystrophy often present with delayed motor milestones, difficulty running or climbing stairs, frequent falls, and a characteristic waddling gait. As the disease progresses, they may develop calf muscle pseudohypertrophy, scoliosis, and cardiac or respiratory complications due to the involvement of the heart and diaphragm muscles. Cognitive difficulties and learning disabilities are also reported in some cases.
The diagnosis of DMD typically involves a combination of clinical evaluation and laboratory tests. Elevated levels of creatine kinase (CK) in the blood are indicative of muscle damage and often prompt further investigation. Genetic testing is the gold standard for confirming mutations in the DMD gene. Muscle biopsy, though less commonly performed today, can reveal dystrophin deficiency. Additional assessments, such as electromyography (EMG) and cardiac evaluations, help monitor disease progression and guide management. Early diagnosis enables timely intervention with corticosteroids, physical therapy, and supportive care aimed at improving quality of life and prolonging mobility.
Duchenne Muscular Dystrophy Epidemiology Segmentation
The Duchenne muscular dystrophy epidemiology section provides insights into the historical and current Duchenne muscular dystrophy patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The Duchenne muscular dystrophy market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Duchenne Muscular Dystrophy
Age-specific Cases of Duchenne Muscular Dystrophy
Ambulatory and Non-ambulatory Cases of Duchenne Muscular Dystrophy
Mutation-specific Cases of Duchenne Muscular Dystrophy
Associated Comorbidities in Duchenne Muscular Dystrophy
Download the report to understand which factors are driving Duchenne muscular dystrophy epidemiology trends @ Duchenne Muscular Dystrophy Treatment Algorithm Duchenne Muscular Dystrophy Report Metrics Details Study Period 2020–2034 Duchenne Muscular Dystrophy Report Coverage 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] Key Duchenne Muscular Dystrophy Companies FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others Key Duchenne Muscular Dystrophy Therapies NS-089/NCNP-02 (brogidirsen), TAS-205 (pizuglanstat), RGX-202, VYONDYS 53, EXONDYS 51, AGAMREE, VILTEPSO, VILTEPSO, and others
Scope of the Duchenne Muscular Dystrophy Market Report Duchenne Muscular Dystrophy Therapeutic Assessment: Duchenne Muscular Dystrophy current marketed and emerging therapies
Duchenne Muscular Dystrophy current marketed and emerging therapies Duchenne Muscular Dystrophy Market Dynamics: Conjoint Analysis of Emerging Duchenne Muscular Dystrophy Drugs
Conjoint Analysis of Emerging Duchenne Muscular Dystrophy Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Duchenne Muscular Dystrophy Market Access and Reimbursement
Discover more about Duchenne muscular dystrophy drugs in development @ Duchenne Muscular Dystrophy Clinical Trials
Table of Contents 1. Duchenne Muscular Dystrophy Market Key Insights 2. Duchenne Muscular Dystrophy Market Report Introduction 3. Duchenne Muscular Dystrophy Market Overview at a Glance 4. Duchenne Muscular Dystrophy Market Executive Summary 5. Disease Background and Overview 6. Duchenne Muscular Dystrophy Treatment and Management 7. Duchenne Muscular Dystrophy Epidemiology and Patient Population 8. Patient Journey 9. Duchenne Muscular Dystrophy Marketed Drugs 10. Duchenne Muscular Dystrophy Emerging Drugs 11. Seven Major Duchenne Muscular Dystrophy Market Analysis 12. Duchenne Muscular Dystrophy Market Outlook 13. Potential of Current and Emerging Therapies 14. KOL Views 15. Unmet Needs 16. SWOT Analysis
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