Duchenne Muscular Dystrophy Market Report 2034: Epidemiology Data, Pipeline Therapies, Latest FDA, EMA, PDMA Approvals by DelveInsight | Sarepta Therapeutics, PTC Therapeutics, Nippon Shinyaku, Santhera Pharmaceuticals/ReveraGen BioPharma, Taiho Pharmaceu

DelveInsight's "Duchenne Muscular Dystrophy Market Insights, 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, France) the United Kingdom and Japan.

To Know in detail about the Duchenne Muscular Dystrophy market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Duchenne Muscular Dystrophy Market Forecast @ https://www.delveinsight.com/report-store/duchenne-muscular-dystrophy-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr


Some of the key facts of the Duchenne Muscular Dystrophy Market Report:
The Duchenne Muscular Dystrophy market size is anticipated to grow with a significant CAGR during the study period (2020-2034).
On November 6, 2024, Cumberland Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) had awarded Ifetroban both Orphan Drug Designation and Rare Pediatric Disease Designation for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The company is actively conducting the FIGHT DMD™ trial, a Phase II multicenter, double-blind, placebo-controlled study designed to assess the pharmacokinetics, safety, and efficacy of once-daily oral Ifetroban in DMD patients. The trial results are anticipated later this year.
In November 2024, Genethon, a prominent gene therapy research organization founded by AFM-Telethon, shared promising results from the Phase 1/2 dose-escalation segment of its international Phase 1/2/3 trial for GNT0004, a gene therapy targeting Duchenne muscular dystrophy (DMD). These findings were presented at the ASGCT Breakthroughs in Muscular Dystrophy conference held on November 19-20, 2024, in Chicago, IL. Building on these encouraging results, Genethon plans to commence pivotal trials in Europe in Q2 2025, followed by trials in the United States shortly afterward.
In September 2024, Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage biotechnology company focused on developing transformative therapies for genetically driven muscle diseases, announced positive new findings from its ongoing Phase 1/2 DELIVER trial. The study, which evaluates DYNE-251 in DMD patients eligible for exon 51 skipping, demonstrated notable dystrophin expression and functional improvements across multiple patient groups.
The Duchenne Muscular Dystrophy (DMD) market in the 7MM was valued at approximately USD 2,150 million in 2023. Significant growth is expected, driven by improved uptake of existing treatments, anticipated launches of one-time gene therapies, and increased awareness.
Key therapeutic strategies for DMD include gene replacement or other mutation-specific genetic therapies to restore dystrophin production, membrane stabilization and upregulation of compensatory proteins, as well as reducing inflammation and enhancing muscle regeneration.
In the US, currently approved DMD treatments include EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), VILTEPSO (viltolarsen), and ELEVIDYS (delandistrogene moxeparvovec). In January 2024, Santhera Pharmaceuticals launched AGAMREE (vamorolone) in Germany for DMD patients aged four and above, marking the company's commercial entry into the biopharma market.
ELEVIDYS, the first FDA-approved gene therapy for DMD, received accelerated approval in June 2023. However, Sarepta's therapy faces scrutiny from ICER over the surrogate endpoints used in the approval process, potentially delaying its full approval.
In the EU4 countries and the UK, steroid therapies dominate, complemented by TRANSLARNA (ataluren) for DMD patients with nonsense mutations. In Japan, VILTEPSO (viltolarsen) is the only approved therapy.
Upcoming therapies from companies such as Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), Capricor (CAP-1002), Italfarmaco (Givinostat), Antisense Therapeutics (ATL1102), and Sarepta Therapeutics (SRP-5051) are anticipated to expand the market significantly. These treatments, alongside a growing prevalence of DMD, are expected to drive market growth in the United States and later in the EU4, the UK, and Japan.
In 2023, the United States had the highest prevalence of Duchenne Muscular Dystrophy (DMD), with approximately 17,200 cases.
Within the EU4 and the UK, the UK reported the highest number of DMD cases, while Spain had the lowest.
In the US, the 5-9-year age group represented the largest proportion of cases in 2023, followed by those aged 10-14 years.
The majority of individuals with Duchenne Muscular Dystrophy are non-ambulatory.
Key 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
Key Duchenne Muscular Dystrophy Therapies: ITF2357 (givinostat), VYONDYS 53, ELEVIDYS (delandistrogene moxeparvovec), and others
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.
Duchenne Muscular Dystrophy Overview
Duchenne Muscular Dystrophy (DMD) is the most common type of muscular dystrophy in childhood, characterized by progressive muscle weakness and degeneration. It is a genetic condition caused by the absence of dystrophin, a protein essential for maintaining muscle cell integrity. Symptom onset typically occurs between the ages of 3 and 5, primarily affecting boys, though rare cases have been reported in girls.
Genetic testing for DMD mutations in a blood sample is crucial, even when the condition is initially confirmed by the absence of dystrophin protein expression in a muscle biopsy. Genetic test results provide essential information for clinical decision-making, including genetic counseling, prenatal diagnosis, and consideration of mutation-specific therapies.
The Duchenne Muscular Dystrophy report offers a comprehensive overview of the disease, including its pathophysiology, diagnostic methods, and detailed treatment algorithms. It also presents a real-world scenario of a patient's journey, from the first appearance of symptoms to diagnosis and the complete treatment process.
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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 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 proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
Diagnosed Prevalence of Duchenne Muscular Dystrophy in Adults
Diagnosed Prevalence of Duchenne Muscular Dystrophy in Pediatrics
Diagnosed Prevalence of Duchenne Muscular Dystrophy by Types
Diagnosed Prevalence of Duchenne Muscular Dystrophy by Location
Diagnosed Prevalence of Duchenne Muscular Dystrophy by Severity
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Duchenne Muscular Dystrophy Marketed Drugs
ELEVIDYS (delandistrogene moxeparvovec): Sarepta Therapeutics
VYONDYS 53: Sarepta Therapeutics
Duchenne Muscular Dystrophy Emerging Drugs
ITF2357 (givinostat): Italfarmaco
Duchenne Muscular Dystrophy Therapies
ITF2357 (givinostat), VYONDYS 53, ELEVIDYS (delandistrogene moxeparvovec), and others
Duchenne Muscular Dystrophy Key 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 Market Outlook
The medical management of Duchenne Muscular Dystrophy (DMD) involves comprehensive care, including cardiac and respiratory support, diet, exercise, bracing, and spinal management. Current treatments combine standard care with emerging therapies such as genetic and cell-based approaches, membrane stabilization, cytoskeletal protein upregulation, and secondary pathway interventions. Supportive care and psychosocial management are also integral.
In the EU4 and the UK, steroid therapies dominate the market alongside TRANSLARNA (ataluren) for patients with nonsense mutations. In Japan, VILTEPSO (viltolarsen) is the only approved treatment.
Sarepta Therapeutics leads the market with three approved therapies and two pipeline candidates, but competition from emerging players like Pfizer, AbbVie, and Italfarmaco is growing. Regulatory approval processes vary significantly across the 7MM (Seven Major Markets), with geographic disparities reflecting differing standards among agencies like the EMA and FDA.
Scope of the Duchenne Muscular Dystrophy Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key 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
Key Duchenne Muscular Dystrophy Therapies: ITF2357 (givinostat), VYONDYS 53, ELEVIDYS (delandistrogene moxeparvovec), and others
Duchenne Muscular Dystrophy Therapeutic Assessment: Duchenne Muscular Dystrophy current marketed and Duchenne Muscular Dystrophy emerging therapies
Duchenne Muscular Dystrophy Market Dynamics: Duchenne Muscular Dystrophy market drivers and Duchenne Muscular Dystrophy market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
Duchenne Muscular Dystrophy Unmet Needs, KOL's views, Analyst's views, Duchenne Muscular Dystrophy Market Access and Reimbursement

To know more about Duchenne Muscular Dystrophy companies working in the treatment market, visit @ Duchenne Muscular Dystrophy Clinical Trials and Therapeutic Assessment @ https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr


Table of Contents
1. Duchenne Muscular Dystrophy Market Report Introduction
2. Executive Summary for Duchenne Muscular Dystrophy
3. SWOT analysis of Duchenne Muscular Dystrophy
4. Duchenne Muscular Dystrophy Patient Share (%) Overview at a Glance
5. Duchenne Muscular Dystrophy Market Overview at a Glance
6. Duchenne Muscular Dystrophy Disease Background and Overview
7. Duchenne Muscular Dystrophy Epidemiology and Patient Population
8. Country-Specific Patient Population of Duchenne Muscular Dystrophy
9. Duchenne Muscular Dystrophy Current Treatment and Medical Practices
10. Duchenne Muscular Dystrophy Unmet Needs
11. Duchenne Muscular Dystrophy Emerging Therapies
12. Duchenne Muscular Dystrophy Market Outlook
13. Country-Wise Duchenne Muscular Dystrophy Market Analysis (2020-2034)
14. Duchenne Muscular Dystrophy Market Access and Reimbursement of Therapies
15. Duchenne Muscular Dystrophy Market Drivers
16. Duchenne Muscular Dystrophy Market Barriers
17. Duchenne Muscular Dystrophy Appendix
18. Duchenne Muscular Dystrophy Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
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Duchenne Muscular Dystrophy Pipeline
"Duchenne Muscular Dystrophy Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Duchenne Muscular Dystrophy market. A detailed picture of the Duchenne Muscular Dystrophy pipeline landscape is provided, which includes the disease overview and Duchenne Muscular Dystrophy treatment guidelines.
Duchenne Muscular Dystrophy Epidemiology
DelveInsight's 'Duchenne Muscular Dystrophy Epidemiology Forecast to 2034' report delivers an in-depth understanding of the disease, historical and forecasted Duchenne Muscular Dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
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