듀셴형 근이영양증 시장 : 세계 및 지역별 - 분석과 예측(2025-2035년)

Global Duchenne Muscular Dystrophy Market, Analysis and Forecast: 2025-2035

Duchenne Muscular Dystrophy is a genetic disorder that primarily affects males and causes progressive muscle weakness and degeneration. It is caused by mutations in the dystrophin gene, which leads to the absence or severe deficiency of dystrophin, a crucial protein responsible for maintaining the structural integrity of muscle fibers. Without dystrophin, muscle cells are unable to function properly and gradually break down. This results in a decline in muscle strength, starting in early childhood and often leading to loss of mobility by adolescence.

Duchenne Muscular Dystrophy typically presents symptoms between the ages of 2 and 3, with common signs including difficulty walking, frequent falls, muscle cramps, and the Gower's sign (a method of using hands and arms to rise from the floor). As the disease progresses, it affects not only the skeletal muscles but also the cardiac and respiratory muscles, leading to heart problems and breathing difficulties. The condition significantly shortens life expectancy, with many individuals passing away in their late teens or early twenties due to respiratory or cardiac complications.

One of the key drivers of the duchenne muscular dystrophy market is the rising prevalence of risk factors such as genetic mutations, family history, and advanced maternal age. Duchenne Muscular Dystrophy is an X-linked recessive genetic disorder, meaning that the primary risk factor is the inheritance of a defective dystrophin gene, which results in the absence or deficiency of dystrophin in muscle cells. Family history plays a crucial role, as the disease is often inherited from carriers, particularly mothers who pass the defective gene to their sons. Additionally, advanced maternal age has been associated with a higher likelihood of genetic mutations that can lead to Duchenne Muscular Dystrophy. Along with these genetic factors, the increasing availability of genetic testing has led to more early diagnoses and greater awareness of Duchenne Muscular Dystrophy, prompting earlier interventions. As the number of diagnosed individuals increases, the demand for innovative treatments, such as gene therapies and exon-skipping drugs, continues to rise, driving the growth of the Duchenne Muscular Dystrophy market. These factors combined are accelerating the market's expansion as they highlight the need for advanced therapeutic options and early intervention strategies.

Despite the growth of the Duchenne Muscular Dystrophy market, several challenges continue to hinder its progress. One of the primary challenges is the high cost of treatment associated with emerging therapies, particularly gene therapies and exon-skipping drugs like Exondys 51 and Vyondys 53. These advanced treatments can be prohibitively expensive, limiting accessibility for many patients, especially in low- and middle-income regions or for those without comprehensive insurance coverage. Additionally, the small patient population for Duchenne Muscular Dystrophy, being a rare disease, means that companies often face challenges in justifying the high cost of research, development, and production of these therapies. Another challenge is the complexity of clinical trials, as Duchenne Muscular Dystrophy affects a genetically diverse patient population, making it difficult to develop universal treatments that work for all patients. Furthermore, the long-term safety and efficacy of new therapies are still being evaluated, and more research is needed to ensure sustained benefits without serious side effects. These factors together create barriers to the widespread adoption of Duchenne Muscular Dystrophy treatments, even as advancements continue to be made.

The global Duchenne Muscular Dystrophy market is highly competitive, with several leading companies driving innovation and market growth such as Sarepta Therapeutics, Pfizer Inc., Wave Life Sciences, Solid Biosciences, and Molecular Partners. These companies are at the forefront of developing groundbreaking therapies, including exon-skipping treatments, gene therapies, and protein replacement therapies to address the underlying genetic causes of Duchenne Muscular Dystrophy. Sarepta Therapeutics, for instance, has pioneered Exondys 51 (eteplirsen) and Vyondys 53 (golodirsen), which utilize exon-skipping technology to improve muscle function in Duchenne Muscular Dystrophy patients. Pfizer Inc. is actively involved with Vyndys 53 and other treatments aimed at genetic mutations in Duchenne Muscular Dystrophy. Wave Life Sciences focuses on advancing therapies that use gene editing and exon-skipping technology. Solid Biosciences and Molecular Partners are also making significant contributions with innovative gene therapies aimed at restoring dystrophin production in muscle cells. These advancements are expected to significantly transform the Duchenne Muscular Dystrophy treatment landscape, addressing the unmet needs of patients and driving substantial market growth.

Market Segmentation:

Segmentation 1: by Region

• North America
• Europe
• Asia-Pacific

The global Duchenne Muscular Dystrophy market is undergoing significant transformation, fueled by emerging trends that are reshaping the way Duchenne Muscular Dystrophy is treated and managed. One of the most prominent trends is the advancement of gene therapies, including innovative approaches like CRISPR gene editing and viral vector-based gene delivery systems, which aim to address the underlying genetic mutations responsible for the disease. These therapies are offering the potential for long-term or even permanent solutions to restore the missing dystrophin protein in muscle cells. Another key trend is the development of exon-skipping drugs, such as Exondys 51 and Vyondys 53, that enable the skipping of faulty exons in the dystrophin gene, allowing to produce a functional, albeit shorter, form of dystrophin.

In addition to these advancements, there is a growing focus on personalized medicine, where treatments are tailored to the specific genetic mutations of individual patients, improving the precision and effectiveness of interventions. The market is also benefiting from regulatory support and orphan drug designations, which help expedite the approval process for new treatments. As early diagnosis through genetic testing becomes more widespread, there is increased demand for early intervention, leading to better outcomes and driving market growth. Furthermore, the establishment of specialized care centers for rare diseases is improving patient access to expert care and the latest treatment options. Together, these trends are not only expanding the Duchenne Muscular Dystrophy market but also offering hope for more effective, personalized, and potentially curative treatments for patients.

Table of Contents

Executive Summary

Scope and Definition

Market/Product Definition

Inclusion and Exclusion

Key Questions Answered

Analysis and Forecast Note

1. Global Duchenne Muscular Dystrophy Market: Industry Outlook

• 1.1 Introduction
• 1.2 Market Trends
• 1.3 Regulatory Framework
• 1.4 Epidemiology Analysis
• 1.5 Clinical Trial Analysis
• 1.6 Market Dynamics
  • 1.6.1 Impact Analysis
  • 1.6.2 Market Drivers
  • 1.6.3 Market Challenges
  • 1.6.4 Market Opportunities

2. Global Duchenne Muscular Dystrophy Market (Region), ($Billion), 2023-2035

• 2.1 North America
  • 2.1.1 Key Findings
  • 2.1.2 Market Dynamics
  • 2.1.3 Market Sizing and Forecast
    • 2.1.3.1 North America Duchenne Muscular Dystrophy Market, by Country
      • 2.1.3.1.1 U.S.
• 2.2 Europe
  • 2.2.1 Key Findings
  • 2.2.2 Market Dynamics
  • 2.2.3 Market Sizing and Forecast
    • 2.2.3.1 Europe Duchenne Muscular Dystrophy Market, by Country
      • 2.2.3.1.1 Germany
      • 2.2.3.1.2 U.K.
      • 2.2.3.1.3 France
      • 2.2.3.1.4 Italy
• 2.3 Asia Pacific
  • 2.3.1 Key Findings
  • 2.3.2 Market Dynamics
  • 2.3.3 Market Sizing and Forecast
    • 2.3.3.1 Asia Pacific Duchenne Muscular Dystrophy Market, by Country
      • 2.3.3.1.1 China
      • 2.3.3.1.2 Japan

3. Global Duchenne Muscular Dystrophy Market: Competitive Landscape and Company Profiles

• 3.1 Key Strategies and Development
  • 3.1.1 Mergers and Acquisitions
  • 3.1.2 Synergistic Activities
  • 3.1.3 Business Expansions and Funding
  • 3.1.4 Product Launches and Approvals
  • 3.1.5 Other Activities
• 3.2 Company Profiles
  • 3.2.1 Sarepta Therapeutics
    • 3.2.1.1 Overview
    • 3.2.1.2 Top Products / Product Portfolio
    • 3.2.1.3 Top Competitors
    • 3.2.1.4 Target Customers/End-Users
    • 3.2.1.5 Key Personnel
    • 3.2.1.6 Analyst View
  • 3.2.2 Pfizer Inc.
    • 3.2.2.1 Overview
    • 3.2.2.2 Top Products / Product Portfolio
    • 3.2.2.3 Top Competitors
    • 3.2.2.4 Target Customers/End-Users
    • 3.2.2.5 Key Personnel
    • 3.2.2.6 Analyst View
  • 3.2.3 Wave Life Sciences
    • 3.2.3.1 Overview
    • 3.2.3.2 Top Products / Product Portfolio
    • 3.2.3.3 Top Competitors
    • 3.2.3.4 Target Customers/End-Users
    • 3.2.3.5 Key Personnel
    • 3.2.3.6 Analyst View
  • 3.2.4 Solid Biosciences
    • 3.2.4.1 Overview
    • 3.2.4.2 Top Products / Product Portfolio
    • 3.2.4.3 Top Competitors
    • 3.2.4.4 Target Customers/End-Users
    • 3.2.4.5 Key Personnel
    • 3.2.4.6 Analyst View
  • 3.2.5 Molecular Partners
    • 3.2.5.1 Overview
    • 3.2.5.2 Top Products / Product Portfolio
    • 3.2.5.3 Top Competitors
    • 3.2.5.4 Target Customers/End-Users
    • 3.2.5.5 Key Personnel
    • 3.2.5.6 Analyst View
  • 3.2.6 Dystrophin Gene Therapy Companies
    • 3.2.6.1 Overview
    • 3.2.6.2 Top Products / Product Portfolio
    • 3.2.6.3 Top Competitors
    • 3.2.6.4 Target Customers/End-Users
    • 3.2.6.5 Key Personnel
    • 3.2.6.6 Analyst View

4. Research Methodology