The Duchenne Muscular Dystrophy market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Duchenne Muscular Dystrophy pipeline products will significantly revolutionize the Duchenne Muscular Dystrophy market dynamics.
DelveInsight’s “Duchenne Muscular Dystrophy Market Insight, Epidemiology and Market Forecast -2034″ report offers an in-depth understanding of the Duchenne Muscular Dystrophy, historical and forecasted epidemiology as well as the Duchenne Muscular Dystrophy market trends in the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.
The Duchenne Muscular Dystrophy market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
To Know in detail about the Duchenne Muscular Dystrophy market outlook, drug uptake, treatment scenario and epidemiology trends, visit Duchenne Muscular Dystrophy Market Insights
Some of the key facts of the Duchenne Muscular Dystrophy Market Report:
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Duchenne Muscular Dystrophy market size was highest in the United States among the 7MM countries
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In September 2025, Satellos Bioscience filed an IND with the U.S. FDA, along with international submissions, to initiate a Phase 2 trial of SAT-3247 in ambulatory children with Duchenne muscular dystrophy (DMD), marking a major milestone for the investigational therapy.
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In August 2025, Keros Therapeutics reported that the FDA granted Orphan Drug Designation to KER-065 for treating Duchenne muscular dystrophy (DMD). The candidate targets disorders stemming from abnormal signaling within the TGF-β protein family.
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In July 2025, the FDA authorized Sarepta to restart shipments of ELEVIDYS (delandistrogene moxeparvovec) for ambulatory Duchenne muscular dystrophy patients.
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In August 2025, Dyne Therapeutics announced that the FDA awarded Breakthrough Therapy Designation to DYNE-251 for DMD patients eligible for exon 51 skipping, based on encouraging results from the DELIVER trial.
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In July 2025, Capricor Therapeutics announced a webinar with Parent Project Muscular Dystrophy (PPMD) to review the status of its BLA for Deramiocel, discuss cardiomyopathy in DMD, and address community questions. The event is scheduled for July 29, 2025.
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In July 2025, Precision BioSciences received Orphan Drug Designation from the FDA for PBGENE-DMD, its gene editing therapy in development for Duchenne muscular dystrophy.
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In July 2025, Avidity Biosciences reported that the FDA granted Breakthrough Therapy Designation to delpacibart zotadirsen (del-zota) for DMD patients with mutations suitable for exon 44 skipping (DMD44). The therapy is being evaluated in the Phase 2 EXPLORE44-OLE™ trial and represents the first of several AOC candidates for DMD.
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In July 2025, Sarepta Therapeutics paused all ELEVIDYS shipments following FDA discussions, due to regulatory concerns around manufacturing and distribution despite prior accelerated approval. The company will work with the agency to address the issues.
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In July 2025, Capricor Therapeutics received a Complete Response Letter from the FDA regarding its BLA for Deramiocel, a cell therapy aimed at treating cardiomyopathy associated with DMD.
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In June 2025, Edgewise Therapeutics shared positive findings from its MESA open-label extension study of sevasemten in Becker and Duchenne muscular dystrophies, with 99% of prior participants (85 patients) enrolled by March 2025.
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In June 2025, Precision BioSciences announced that PBGENE-DMD, its ARCUS®-based in vivo gene editing therapy for DMD, received Rare Pediatric Disease Designation from the FDA.
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In June 2025, Sarepta Therapeutics issued a safety update on ELEVIDYS after two acute liver failure cases in non-ambulatory DMD patients. Shipments for this group are paused until an improved immunosuppressive protocol is approved.
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In May 2025, Capricor Therapeutics completed its mid-cycle FDA review for the Deramiocel BLA. The agency reported no major issues and confirmed that the application remains on track for the August 31, 2025 PDUFA date, with an advisory committee meeting planned.
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In March 2025, the FDA accepted Capricor’s BLA for Deramiocel, granting priority review to expedite a decision for the DMD cardiomyopathy therapy.
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In January 2025, Capricor Therapeutics submitted its BLA to the FDA seeking full approval of Deramiocel, its investigational cell therapy for DMD-related cardiomyopathy.
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Duchenne Muscular Dystrophy Companies: Sarepta Therapeutics, PTC Therapeutics, Nippon Shinyaku, Santhera Pharmaceuticals/ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Capricor, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, and others
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Duchenne Muscular Dystrophy Therapies: ELEVIDYS (delandistrogene moxeparvovec), VYONDYS 53, AMONDYS 45, ITF2357 (givinostat), TAS-205, and others.
Duchenne Muscular Dystrophy Overview
Duchenne muscular dystrophy (DMD) is a severe, progressive genetic disorder that primarily affects males, causing progressive muscle weakness and wasting due to the absence of the protein dystrophin. The condition affects skeletal, heart, and lung muscles and significantly shortens life expectancy, though advances in care have improved prognosis.
Duchenne Muscular Dystrophy Market
The dynamics of the Duchenne Muscular Dystrophy market are anticipated to change in the coming years owing to the expected launch of emerging therapies and others during the forecasted period 2025-2034.
Duchenne Muscular Dystrophy Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and the views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Duchenne Muscular Dystrophy Epidemiology Segmentation:
The Duchenne Muscular Dystrophy market report offers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
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Total Prevalent Cases of Duchenne Muscular Dystrophy in the 7MM
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Age-specific Cases of Duchenne Muscular Dystrophy in the 7MM
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Ambulatory and Non-ambulatory Cases of Duchenne Muscular Dystrophy in the 7MM
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Mutation-specific Cases of Duchenne Muscular Dystrophy in the 7MM
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Associated Comorbidities in Duchenne Muscular Dystrophy in the 7MM
Download the report to understand which factors are driving Duchenne Muscular Dystrophy epidemiology trends @ Duchenne Muscular Dystrophy Epidemiological Insights
Duchenne Muscular Dystrophy Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Duchenne Muscular Dystrophy market or expected to get launched during the study period. The analysis covers Duchenne Muscular Dystrophy market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Duchenne Muscular Dystrophy Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
To know more about Duchenne Muscular Dystrophy treatment, visit @ Duchenne Muscular Dystrophy Medications
Duchenne Muscular Dystrophy Therapies and Key Companies
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ELEVIDYS (delandistrogene moxeparvovec): Sarepta Therapeutics
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VYONDYS 53: Sarepta Therapeutics
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AMONDYS 45: Sarepta Therapeutics, and others
Duchenne Muscular Dystrophy Market Drivers
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Rising global prevalence of DMD, increasing the need for earlier diagnosis and long-term management.
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Advances in gene therapy and gene-editing technologies (e.g., AAV therapies, exon skipping, CRISPR) accelerating pipeline growth.
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Regulatory incentives such as Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations supporting rapid development.
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Strong clinical pipeline with multiple therapies in Phase 2/3 trials targeting both ambulatory and non-ambulatory populations.
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High unmet medical need, as existing treatments offer limited functional improvement or disease modification.
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Growing investments and partnerships among biotech companies, academic centers, and patient advocacy groups.
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Improved understanding of DMD pathology, enabling targeted therapeutic approaches (e.g., micro-dystrophin, TGF-β modulation).
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Favorable reimbursement pathways for rare disease therapies, supporting premium pricing.
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Increasing newborn screening initiatives aiding early detection and early therapeutic intervention.
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Rising awareness and advocacy efforts from groups like PPMD, MDA, and CureDuchenne driving patient engagement and trial participation.
Duchenne Muscular Dystrophy Market Barriers
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High treatment costs for gene therapies and advanced biologics, limiting accessibility and payer acceptance.
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Complex manufacturing and scalability challenges, especially for gene therapies and cell-based treatments.
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Regulatory hurdles related to safety, long-term efficacy, and durability of response for novel modalities.
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Safety concerns, including liver toxicity, immune reactions, and vector-related adverse events.
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Limited real-world evidence due to small patient populations and variability in disease progression.
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Challenges in patient recruitment, especially for mutation-specific therapies with narrow eligible populations.
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Short therapeutic windows, as many treatments are more effective in early or ambulatory stages.
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Heterogeneous genetic mutations complicating development of broadly applicable therapies.
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Competition within the pipeline, with many companies targeting similar mechanisms (e.g., exon skipping, micro-dystrophin).
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Reimbursement and pricing pressures, as payers scrutinize high-cost rare disease treatments for value and durability.
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Long-term follow-up requirements, increasing trial duration and overall development costs.
Scope of the Duchenne Muscular Dystrophy Market Report
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Study Period: 2020–2034
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Coverage: 7MM [The United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]
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Key Duchenne Muscular Dystrophy Companies: Companies: Sarepta Therapeutics, PTC Therapeutics, Nippon Shinyaku, Santhera Pharmaceuticals/ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Capricor, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, and others
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Key Duchenne Muscular Dystrophy Therapies: ELEVIDYS (delandistrogene moxeparvovec), VYONDYS 53, AMONDYS 45, ITF2357 (givinostat), TAS-205, and others.
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Duchenne Muscular Dystrophy Therapeutic Assessment: Duchenne Muscular Dystrophy current marketed and Duchenne Muscular Dystrophy emerging therapies
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Duchenne Muscular Dystrophy Market Dynamics: Duchenne Muscular Dystrophy market drivers and Duchenne Muscular Dystrophy market barriers
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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
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Duchenne Muscular Dystrophy Unmet Needs, KOL’s views, Analyst’s views, Duchenne Muscular Dystrophy Market Access and Reimbursement
Discover more about therapies set to grab major Duchenne Muscular Dystrophy market share @ Duchenne Muscular Dystrophy Treatment Landscape
Table of Contents
1. Key Insights
2. Report Introduction
3. Executive Summary
4. Duchenne Muscular Dystrophy Market Overview at a Glance
5. Epidemiology and Market Forecast Methodology
6. Key Events
7. Duchenne Muscular Dystrophy Background and Overview
8. Duchenne Muscular Dystrophy Treatment
9. Duchenne Muscular Dystrophy Epidemiology and Patient Population in the 7MM
10. Duchenne Muscular Dystrophy Patient Journey
11. Duchenne Muscular Dystrophy Marketed Drug
12. Duchenne Muscular Dystrophy Emerging Drugs
13. Duchenne Muscular Dystrophy: 7MM Analysis
14. Unmet needs
15. SWOT Analysis
16. KOL Views
17. Market Access and Reimbursement
18. Appendix
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence.
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