Duchenne氏肌肉失養症市場 - 全球及區域 - 分析與預測（2025-2035）

Duchenne氏肌肉失養症是一種主要影響男性的遺傳性疾病，會導致肌肉進行性無力和退化。

肌肉營養不良症是由Dystrophin蛋白基因突變引起的，該基因突變會導致Dystrophin（一種維持肌纖維結構完整性的關鍵蛋白質）的缺失或嚴重缺乏。缺乏Dystrophin，肌肉細胞無法正常運作，逐漸衰竭。結果是，從兒童早期開始出現肌肉無力，並經常在青春期喪失運動技能。

Duchenne氏肌肉失養症通常發病於2至3歲之間，常見症狀包括行走困難、頻繁跌倒、肌肉痙攣和高爾氏徵（一種用手和手臂從地面站起的動作）。隨著病情進展，它不僅會影響骨骼肌，還會影響心肌和呼吸肌肉，導致心臟問題和呼吸困難。這種疾病會顯著縮短壽命，許多人在十幾歲或二十出頭時就因呼吸和心臟併發症死亡。

Duchenne氏肌肉失養症市場的主要驅動力之一是基因突變、家族病史和高齡產婦等風險因素的日益普及。Duchenne氏肌肉失養症是一種 X 連鎖隱性遺傳疾病，主要風險因子是Dystrophin基因的遺傳缺陷。家族病史起著關鍵作用，因為這種疾病通常會遺傳給帶因者，尤其是母親傳給兒子。此外，母親年齡越大，導致Duchenne氏肌肉失養症的基因突變的幾率就越高。這些遺傳因素，加上基因檢測的普及，使得早期診斷成為可能，提高了人們對Duchenne氏肌肉失養症的認知，並鼓勵早期介入。隨著確診患者數量的增加，對基因療法和外顯子跳躍藥物等創新治療的需求也在不斷成長，從而推動了Duchenne氏肌肉失養症市場的成長。這些因素共同凸顯了對先進治療方案和早期療育策略的需求，加速了市場擴張。

儘管Duchenne氏肌肉失養症市場正在成長，但仍有幾個挑戰阻礙著它的發展。其中一個主要挑戰是新治療方法治療費用高昂，尤其是基因療法和外顯子跳躍藥物，如Exondys 51和Vyondys 53。這些先進療法價格高昂，限制了許多患者獲得治療的機會，特別是中低收入地區和沒有全面保險覆蓋的患者。此外，Duchenne氏肌肉失養症是一種罕見疾病，這意味著患者很少，公司經常面臨挑戰，難以證明這些治療方法的研究、開發和製造成本高。另一個挑戰是，Duchenne氏肌肉失養症影響到基因多樣化的患者群體，因此很難開發出適用於所有患者的通用治療方法，並使臨床試驗複雜化。此外，新治療方法的長期安全性和有效性仍在評估中，需要進一步研究以確保持續療效而沒有嚴重的副作用。儘管Duchenne氏肌肉失養症的治療不斷取得進展，但這些因素共同阻礙了疾病的廣泛應用。

全球Duchenne氏肌肉失養症市場競爭激烈，Sarepta Therapeutics、輝瑞公司、Wave Life Sciences、Solid Biosciences 和 Molecular Partners 等主要企業推動創新和市場成長。這些公司處於開發突破性治療方法的前沿，例如外顯子跳躍療法、基因療法和蛋白質替代療法，以解決Duchenne氏肌肉失養症的潛在遺傳原因。例如，Sarepta Therapeutics 開發了 Exondys 51 (eteplirsen) 和 Vyondys 53 (gorodirsen)，它們利用外顯子跳躍技術改善Duchenne氏肌肉失養症患者的肌肉功能。輝瑞正積極致力於針對 Vyndys 53 和其他Duchenne氏肌肉失養症基因突變的療法。 Wave Life Sciences 專注於使用基因編輯和外顯子跳躍技術來開發治療方法。 Solid Biosciences 和 Molecular Partners 也憑藉著旨在恢復肌肉細胞中肌肉Dystrophin生成的創新基因療法藥物做出了重大貢獻。這些進展預計將徹底改變Duchenne氏肌肉失養症的治療格局，滿足患者尚未滿足的需求，並推動市場顯著成長。

市場區隔

細分一：按地區

• 北美洲
• 歐洲
• 亞太地區

全球Duchenne氏肌肉失養症市場正經歷重大變革時期期，推動其發展的新興趨勢正在重塑Duchenne氏肌肉失養症的治療和管理方式。最突出的趨勢之一是基因治療的進步，包括 CRISPR 基因編輯和基於病毒載體的基因傳遞系統等創新方法，旨在解決導致疾病的潛在基因突變。這些治療方法為恢復肌肉細胞中缺失的Dystrophin蛋白提供了長期甚至永久的解決方案。另一個主要趨勢是外顯子跳躍藥物（如 Exondys 51 和 Vyondys 53）的開發，這些藥物可以跳過肌肉營養不良Dystrophin基因中有缺陷的外顯子，從而產生更短但有功能性的Dystrophin。

除了這些進展之外，個人化醫療也越來越受到關注，即根據個別患者的特定基因突變量身定做治療方案，從而提高介入的精確度和有效性。市場也受益於監管支持，加快了新治療方法和孤兒藥指定的核准流程。隨著透過基因檢測進行早期診斷變得越來越普遍，對早期療育的需求也在增加，從而帶來更好的結果，推動市場成長。此外，罕見疾病專科治療中心的建立正在改善患者獲得專家護理和最新治療方案的機會。這些趨勢不僅擴大了Duchenne氏肌肉失養症市場，也為患者帶來了更有效、個人化和潛在治癒治療方法的希望。

本報告研究了全球Duchenne氏肌肉失養症市場，並概述了市場以及區域趨勢和公司概況。

目錄

執行摘要

第1章 全球Duchenne氏肌肉失養症市場：產業展望

• 介紹
• 市場趨勢
• 法律規範
• 流行病學分析
• 臨床試驗分析
• 市場動態

2. 2023-2035 年全球Duchenne氏肌肉失養症市場（按地區）

• 北美洲
• 歐洲
• 亞太地區

3. 全球Duchenne氏肌肉失養症市場：競爭格局與公司概況

• 關鍵策略和發展
  • 合併與收購
  • 協同活動
  • 業務擴展和資金籌措
  • 產品發布和核准
  • 其他活動
• 公司簡介
  • Sarepta Therapeutics
  • Pfizer Inc.
  • Wave Life Sciences
  • Solid Biosciences
  • Molecular Partners
  • Dystrophin Gene Therapy Companies

第4章調查方法

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

List of Figures

• Figure: Global Duchenne Muscular Dystrophy Market (by Region), $Billion, 2024 and 2035
• Figure: Global Duchenne Muscular Dystrophy Market Key Trends, Analysis

List of Tables

• Table: Global Duchenne Muscular Dystrophy Market Dynamics, Impact Analysis
• Table: Global Duchenne Muscular Dystrophy Market (by Region), $Billion, 2024-2035