듀셴형 근이영양증 : 세계 시장 점유율과 순위, 총판매량 및 수요 예측(2025-2031년)

The global market for Duchenne Muscular Dystrophy was estimated to be worth US$ 2067 million in 2024 and is forecast to a readjusted size of US$ 4449 million by 2031 with a CAGR of 13.1% during the forecast period 2025-2031.

Duchenne Muscular Dystrophy is an x-linked genetic disorder that affects mostly boys. In Duchenne, boys begin to show signs of muscle weakness as early as two to five years of age. The disease gradually weakens the skeletal or voluntary muscles in the arms, legs and trunk. Due to progressive muscle weakness, Duchenne patients are often wheelchair bound between the ages of seven and 13 years old. At a later stage, the boys' respiratory and cardiac muscles are also affected and for most boys, respiratory and cardiac failure are major causes of death, often prevalent by the age of 20.

Global Duchenne Muscular Dystrophy key players include Sarepta Therapeutics, PTC Therapeutics, etc. North America is the largest market, with a share over 70%, followed by Europe, and Japan, both have a share over 25 percent. In terms of product, Exondys 51 is the largest segment, with a share over 55%. And in terms of application, the largest application is Hospitals, followed by Clinics, Home Care, etc.

The Duchenne muscular dystrophy (DMD) market is primarily driven by the following factors:

1. Disease Burden and Epidemiological Data

• 1.1 Incidence and Patient Population

Global Data: DMD is a rare, X-linked genetic disease with a global incidence of approximately 6 cases per 100,000 people, with the majority of patients being male (due to recessive inheritance of the X chromosome, females require two defective copies to develop the disease).

China Data: The incidence of DMD in China is approximately 1 in 3,853, with approximately 60,000 to 70,000 patients nationwide. The majority of patients are children and adolescents, with the peak incidence occurring between the ages of 3 and 5 years.

Regional Distribution: Europe (Sweden and Norway), North America (the United States and Canada), and China are the most prevalent regions, with the Asia-Pacific region (such as Australia and Japan) contributing approximately 30% of clinical trials.

• 1.2 Disease Characteristics and Impact

Symptoms and Prognosis: Patients typically experience delayed motor development (e.g., difficulty sitting and walking) at 2-3 years of age, lose the ability to walk around 12 years of age, and ultimately die from respiratory or cardiopulmonary failure. The average life expectancy is approximately 40 years. Complications: Approximately 30% of patients experience mild intellectual disability, and skeletal deformities (such as scoliosis) and muscle atrophy significantly impact their quality of life.

2. Treatment Advances and Drugs in Development

• 2.1 Marketed Drugs

Corticosteroids: Corticosteroids such as deflazacort (Emflaza) and vamorolone are traditional treatments that slow disease progression through their anti-inflammatory effects, but they can also cause side effects (such as weight gain and growth suppression).

Exon-skipping therapy: Antisense oligonucleotides (ASOs) are used to skip the mutated exon, restoring some dystrophin function. Approved drugs include:

Exondys 51 (exon 51 skipping, affecting 13% of DMD patients), Vyondys 53 (exon 53 skipping, affecting 8%), and Amondys 45 (exon 45 skipping).

Viltepsiso (exon 53 skipping, manufactured by Nippon Shinyaku Co., Ltd.). Gene Therapy: Elevidys (SRP-9001) is the first approved gene therapy for DMD. It delivers the dystrophin gene via an AAV vector. Its indication will be expanded to patients aged 4 years and older (regardless of ambulatory status) in 2024.

• 2.2 Drugs and Technologies in Development

Gene Therapy: Several companies (such as Sarepta Therapeutics and Roche) are advancing gene therapies to address the underlying genetic defect.

Cell Therapy and Gene Editing: Gene editing tools such as CRISPR technology have shown promise in preclinical studies but have yet to enter large-scale application.

HDAC inhibitors: For example, givinostat (Duvyzat) inhibits histone deacetylases to slow muscle degeneration and was approved by the FDA in 2024 for patients aged 6 years and older.

3. Policy Support and Orphan Drug Designation

• 3.1 Global Policy Differences

US: The FDA has approved several DMD drugs (such as Exondys 51 and Elevidys) through accelerated approval, but requires confirmatory trials to verify efficacy. The European Union (EU) has refused to renew approval for some drugs (such as Exondys 51), while the UK's MHRA has retained access to some drugs, reflecting regional policy differences.

China has included DMD in its first list of rare diseases, providing dedicated funding to support research and development, and promoting priority review and reimbursement of new drugs (such as valmorolone).

• 3.2 Orphan Drug Incentives

Tax Incentives and Expedited Approval: Most countries use orphan drug designation (such as the US Orphan Drug Act) to provide tax breaks, research and development subsidies, and fast-track approvals, reducing R&D risks for companies.

Safety Net Programs: Some countries (such as France and Germany) waive economic analysis for drugs with minimal budgetary impact, accelerating patient access.

4. Patient Needs and Organizational Activities

• 4.1 Patient Demands and Quality of Life

Evolving Treatment Needs: Patients and families have a strong demand for treatments that reduce the side effects of traditional hormones (such as newer dissociative steroids like valmorolone), driving the development of innovative therapies. Raising Disease Awareness: Patient organizations (such as the Duchenne Muscular Dystrophy Association) are raising public awareness of DMD through advocacy, advocating for more treatment options and medical insurance coverage.

• 4.2 Optimizing Clinical Management

Multidisciplinary Integrated Management: For example, Beijing Children's Hospital has established a multidisciplinary DMD team (including neurology, rehabilitation, and cardiology) to provide personalized treatment throughout the lifespan.

Early Diagnosis and Intervention: Motor function assessments, muscle enzyme profiles (creatine kinase elevation of 20-200 times), and genetic testing are used to achieve early diagnosis and optimize the therapeutic window.

5. Capital Investment and R&D Trends

• 5.1 Pharmaceutical Company Strategies

International giants: Sarepta Therapeutics (leading in exon skipping therapies), Roche (gene therapy collaborations), Italfarmaco (HDAC inhibitors), and other companies are driving the development of innovative therapies.

Chinese companies: Shufang Pharmaceuticals (valmorolone launched domestically), New Sprout Gene, and Jinweike are investing in gene therapy, bringing new drugs to market through collaborative acquisitions or independent R&D. 5.2 Venture Capital and M&A

Capital Activity: In 2023-2024, several international pharmaceutical companies entered into collaborations with Chinese DMD R&D companies (e.g., Cranbury Pharmaceuticals introduced generic versions of deflazacort), promoting the development of innovative therapies such as gene therapy.

Market Forecast: China's DMD drug market is expected to continue to grow from 2024 to 2030, with innovative therapies (e.g., gene therapy) dominating the market and driving increased market penetration.

6. Technological Advances and Future Directions

• 6.1 Gene Therapy Breakthroughs

Elevidys' Milestone: As the first DMD gene therapy, Elevidys addresses the underlying genetic cause with a one-time treatment. Despite challenges with immunity and contraindications, it represents a future direction.

Long-Term Efficacy Verification: Confirmatory trials (e.g., the EMBARK trial) will continuously track patient functional improvements (e.g., North Star Walk Assessment, Time to Stand), verifying the durability of gene therapy.

• 6.2 Personalized Treatment and Digitalization

Precision Medicine: Developing personalized treatment plans based on the patient's genetic mutation type (e.g., exon skipping therapy) improves efficacy. Digital healthcare applications: Technologies such as online diagnosis and treatment, remote monitoring (such as the "Green Channel" system at Beijing Children's Hospital) optimize patient management and enhance the medical experience.

The growth of the Duchenne muscular dystrophy (DMD) market is primarily driven by the disease burden, treatment advances (particularly gene therapy), policy support, escalating patient needs, and capital investment. In the future, with the approval of more innovative therapies and expanded medical insurance coverage, market demand will be further unleashed, and cutting-edge technologies such as gene therapy are expected to become mainstream treatments.

This report aims to provide a comprehensive presentation of the global market for Duchenne Muscular Dystrophy, focusing on the total sales revenue, key companies market share and ranking, together with an analysis of Duchenne Muscular Dystrophy by region & country, by Type, and by Application.

The Duchenne Muscular Dystrophy market size, estimations, and forecasts are provided in terms of sales revenue ($ millions), considering 2024 as the base year, with history and forecast data for the period from 2020 to 2031. With both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding Duchenne Muscular Dystrophy.

Market Segmentation

By Company

• Sarepta Therapeutics
• PTC Therapeutics
• Pfizer
• Bristol-Myers Squibb
• Italfarmaco
• Santhera Pharmaceuticals

Segment by Type

• Exondys 51
• Emflaza
• Translarna

Segment by Application

• Hospitals
• Clinics
• Home Care

By Region

• North America
  • United States
  • Canada
• Asia-Pacific
  • China
  • Japan
  • South Korea
  • Southeast Asia
  • India
  • Australia
  • Rest of Asia-Pacific
• Europe
  • Germany
  • France
  • U.K.
  • Italy
  • Netherlands
  • Nordic Countries
  • Rest of Europe
• Latin America
  • Mexico
  • Brazil
  • Rest of Latin America
• Middle East & Africa
  • Turkey
  • Saudi Arabia
  • UAE
  • Rest of MEA

Chapter Outline

Chapter 1: Introduces the report scope of the report, global total market size. This chapter also provides the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by manufacturers in the industry, and the analysis of relevant policies in the industry.

Chapter 2: Detailed analysis of Duchenne Muscular Dystrophy company competitive landscape, revenue market share, latest development plan, merger, and acquisition information, etc.

Chapter 3: Provides the analysis of various market segments by Type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.

Chapter 4: Provides the analysis of various market segments by Application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.

Chapter 5: Revenue of Duchenne Muscular Dystrophy in regional level. It provides a quantitative analysis of the market size and development potential of each region and introduces the market development, future development prospects, market space, and market size of each country in the world.

Chapter 6: Revenue of Duchenne Muscular Dystrophy in country level. It provides sigmate data by Type, and by Application for each country/region.

Chapter 7: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product revenue, gross margin, product introduction, recent development, etc.

Chapter 8: Analysis of industrial chain, including the upstream and downstream of the industry.

Chapter 9: Conclusion.

Table of Contents

1 Market Overview

• 1.1 Duchenne Muscular Dystrophy Product Introduction
• 1.2 Global Duchenne Muscular Dystrophy Market Size Forecast (2020-2031)
• 1.3 Duchenne Muscular Dystrophy Market Trends & Drivers
  • 1.3.1 Duchenne Muscular Dystrophy Industry Trends
  • 1.3.2 Duchenne Muscular Dystrophy Market Drivers & Opportunity
  • 1.3.3 Duchenne Muscular Dystrophy Market Challenges
  • 1.3.4 Duchenne Muscular Dystrophy Market Restraints
• 1.4 Assumptions and Limitations
• 1.5 Study Objectives
• 1.6 Years Considered

2 Competitive Analysis by Company

• 2.1 Global Duchenne Muscular Dystrophy Players Revenue Ranking (2024)
• 2.2 Global Duchenne Muscular Dystrophy Revenue by Company (2020-2025)
• 2.3 Key Companies Duchenne Muscular Dystrophy Manufacturing Base Distribution and Headquarters
• 2.4 Key Companies Duchenne Muscular Dystrophy Product Offered
• 2.5 Key Companies Time to Begin Mass Production of Duchenne Muscular Dystrophy
• 2.6 Duchenne Muscular Dystrophy Market Competitive Analysis
  • 2.6.1 Duchenne Muscular Dystrophy Market Concentration Rate (2020-2025)
  • 2.6.2 Global 5 and 10 Largest Companies by Duchenne Muscular Dystrophy Revenue in 2024
  • 2.6.3 Global Top Companies by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in Duchenne Muscular Dystrophy as of 2024)
• 2.7 Mergers & Acquisitions, Expansion

3 Segmentation by Type

• 3.1 Introduction by Type
  • 3.1.1 Exondys 51
  • 3.1.2 Emflaza
  • 3.1.3 Translarna
• 3.2 Global Duchenne Muscular Dystrophy Sales Value by Type
  • 3.2.1 Global Duchenne Muscular Dystrophy Sales Value by Type (2020 VS 2024 VS 2031)
  • 3.2.2 Global Duchenne Muscular Dystrophy Sales Value, by Type (2020-2031)
  • 3.2.3 Global Duchenne Muscular Dystrophy Sales Value, by Type (%) (2020-2031)

4 Segmentation by Application

• 4.1 Introduction by Application
  • 4.1.1 Hospitals
  • 4.1.2 Clinics
  • 4.1.3 Home Care
• 4.2 Global Duchenne Muscular Dystrophy Sales Value by Application
  • 4.2.1 Global Duchenne Muscular Dystrophy Sales Value by Application (2020 VS 2024 VS 2031)
  • 4.2.2 Global Duchenne Muscular Dystrophy Sales Value, by Application (2020-2031)
  • 4.2.3 Global Duchenne Muscular Dystrophy Sales Value, by Application (%) (2020-2031)

5 Segmentation by Region

• 5.1 Global Duchenne Muscular Dystrophy Sales Value by Region
  • 5.1.1 Global Duchenne Muscular Dystrophy Sales Value by Region: 2020 VS 2024 VS 2031
  • 5.1.2 Global Duchenne Muscular Dystrophy Sales Value by Region (2020-2025)
  • 5.1.3 Global Duchenne Muscular Dystrophy Sales Value by Region (2026-2031)
  • 5.1.4 Global Duchenne Muscular Dystrophy Sales Value by Region (%), (2020-2031)
• 5.2 North America
  • 5.2.1 North America Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 5.2.2 North America Duchenne Muscular Dystrophy Sales Value by Country (%), 2024 VS 2031
• 5.3 Europe
  • 5.3.1 Europe Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 5.3.2 Europe Duchenne Muscular Dystrophy Sales Value by Country (%), 2024 VS 2031
• 5.4 Asia Pacific
  • 5.4.1 Asia Pacific Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 5.4.2 Asia Pacific Duchenne Muscular Dystrophy Sales Value by Region (%), 2024 VS 2031
• 5.5 South America
  • 5.5.1 South America Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 5.5.2 South America Duchenne Muscular Dystrophy Sales Value by Country (%), 2024 VS 2031
• 5.6 Middle East & Africa
  • 5.6.1 Middle East & Africa Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 5.6.2 Middle East & Africa Duchenne Muscular Dystrophy Sales Value by Country (%), 2024 VS 2031

6 Segmentation by Key Countries/Regions

• 6.1 Key Countries/Regions Duchenne Muscular Dystrophy Sales Value Growth Trends, 2020 VS 2024 VS 2031
• 6.2 Key Countries/Regions Duchenne Muscular Dystrophy Sales Value, 2020-2031
• 6.3 United States
  • 6.3.1 United States Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 6.3.2 United States Duchenne Muscular Dystrophy Sales Value by Type (%), 2024 VS 2031
  • 6.3.3 United States Duchenne Muscular Dystrophy Sales Value by Application, 2024 VS 2031
• 6.4 Europe
  • 6.4.1 Europe Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 6.4.2 Europe Duchenne Muscular Dystrophy Sales Value by Type (%), 2024 VS 2031
  • 6.4.3 Europe Duchenne Muscular Dystrophy Sales Value by Application, 2024 VS 2031
• 6.5 China
  • 6.5.1 China Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 6.5.2 China Duchenne Muscular Dystrophy Sales Value by Type (%), 2024 VS 2031
  • 6.5.3 China Duchenne Muscular Dystrophy Sales Value by Application, 2024 VS 2031
• 6.6 Japan
  • 6.6.1 Japan Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 6.6.2 Japan Duchenne Muscular Dystrophy Sales Value by Type (%), 2024 VS 2031
  • 6.6.3 Japan Duchenne Muscular Dystrophy Sales Value by Application, 2024 VS 2031
• 6.7 South Korea
  • 6.7.1 South Korea Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 6.7.2 South Korea Duchenne Muscular Dystrophy Sales Value by Type (%), 2024 VS 2031
  • 6.7.3 South Korea Duchenne Muscular Dystrophy Sales Value by Application, 2024 VS 2031
• 6.8 Southeast Asia
  • 6.8.1 Southeast Asia Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 6.8.2 Southeast Asia Duchenne Muscular Dystrophy Sales Value by Type (%), 2024 VS 2031
  • 6.8.3 Southeast Asia Duchenne Muscular Dystrophy Sales Value by Application, 2024 VS 2031
• 6.9 India
  • 6.9.1 India Duchenne Muscular Dystrophy Sales Value, 2020-2031
  • 6.9.2 India Duchenne Muscular Dystrophy Sales Value by Type (%), 2024 VS 2031
  • 6.9.3 India Duchenne Muscular Dystrophy Sales Value by Application, 2024 VS 2031

7 Company Profiles

• 7.1 Sarepta Therapeutics
  • 7.1.1 Sarepta Therapeutics Profile
  • 7.1.2 Sarepta Therapeutics Main Business
  • 7.1.3 Sarepta Therapeutics Duchenne Muscular Dystrophy Products, Services and Solutions
  • 7.1.4 Sarepta Therapeutics Duchenne Muscular Dystrophy Revenue (US$ Million) & (2020-2025)
  • 7.1.5 Sarepta Therapeutics Recent Developments
• 7.2 PTC Therapeutics
  • 7.2.1 PTC Therapeutics Profile
  • 7.2.2 PTC Therapeutics Main Business
  • 7.2.3 PTC Therapeutics Duchenne Muscular Dystrophy Products, Services and Solutions
  • 7.2.4 PTC Therapeutics Duchenne Muscular Dystrophy Revenue (US$ Million) & (2020-2025)
  • 7.2.5 PTC Therapeutics Recent Developments
• 7.3 Pfizer
  • 7.3.1 Pfizer Profile
  • 7.3.2 Pfizer Main Business
  • 7.3.3 Pfizer Duchenne Muscular Dystrophy Products, Services and Solutions
  • 7.3.4 Pfizer Duchenne Muscular Dystrophy Revenue (US$ Million) & (2020-2025)
  • 7.3.5 Pfizer Recent Developments
• 7.4 Bristol-Myers Squibb
  • 7.4.1 Bristol-Myers Squibb Profile
  • 7.4.2 Bristol-Myers Squibb Main Business
  • 7.4.3 Bristol-Myers Squibb Duchenne Muscular Dystrophy Products, Services and Solutions
  • 7.4.4 Bristol-Myers Squibb Duchenne Muscular Dystrophy Revenue (US$ Million) & (2020-2025)
  • 7.4.5 Bristol-Myers Squibb Recent Developments
• 7.5 Italfarmaco
  • 7.5.1 Italfarmaco Profile
  • 7.5.2 Italfarmaco Main Business
  • 7.5.3 Italfarmaco Duchenne Muscular Dystrophy Products, Services and Solutions
  • 7.5.4 Italfarmaco Duchenne Muscular Dystrophy Revenue (US$ Million) & (2020-2025)
  • 7.5.5 Italfarmaco Recent Developments
• 7.6 Santhera Pharmaceuticals
  • 7.6.1 Santhera Pharmaceuticals Profile
  • 7.6.2 Santhera Pharmaceuticals Main Business
  • 7.6.3 Santhera Pharmaceuticals Duchenne Muscular Dystrophy Products, Services and Solutions
  • 7.6.4 Santhera Pharmaceuticals Duchenne Muscular Dystrophy Revenue (US$ Million) & (2020-2025)
  • 7.6.5 Santhera Pharmaceuticals Recent Developments

8 Industry Chain Analysis

• 8.1 Duchenne Muscular Dystrophy Industrial Chain
• 8.2 Duchenne Muscular Dystrophy Upstream Analysis
  • 8.2.1 Key Raw Materials
  • 8.2.2 Raw Materials Key Suppliers
  • 8.2.3 Manufacturing Cost Structure
• 8.3 Midstream Analysis
• 8.4 Downstream Analysis (Customers Analysis)
• 8.5 Sales Model and Sales Channels
  • 8.5.1 Duchenne Muscular Dystrophy Sales Model
  • 8.5.2 Sales Channel
  • 8.5.3 Duchenne Muscular Dystrophy Distributors

9 Research Findings and Conclusion

10 Appendix

• 10.1 Research Methodology
  • 10.1.1 Methodology/Research Approach
    • 10.1.1.1 Research Programs/Design
    • 10.1.1.2 Market Size Estimation
    • 10.1.1.3 Market Breakdown and Data Triangulation
  • 10.1.2 Data Source
    • 10.1.2.1 Secondary Sources
    • 10.1.2.2 Primary Sources
• 10.2 Author Details
• 10.3 Disclaimer