全球杜氏肌肉营养不良症 (DMD) 治疗市场规模（按治疗类型、治疗环境、地区、范围和预测）

杜氏肌肉营养不良症 (DMD) 治疗市场规模及预测

杜氏肌肉营养不良症 (DMD) 治疗市场规模在 2024 年价值 7.2279 亿美元，预计到 2031 年将达到 19.0524 亿美元，2024 年至 2031 年的复合年增长率为 12.88%。杜氏肌肉营养不良症 (DMD) 治疗的主要目标是控制症状、减缓病情进展和改善生活品质。儘管有潜在的副作用，但通常使用泼尼鬆或地夫可特等皮质类固醇来帮助维持肌肉力量和功能。物理治疗和个人化的运动计画在维持活动能力和预防挛缩方面发挥重要作用。

除了皮质类固醇外，艾地苯醌和依替普利生等药物也许能够解决与 DMD 相关的某些课题。基因疗法和外显子跳跃药物等有前景的新疗法针对的是 DMD 的遗传基础，并可能带来长期改善。

CRISPR-Cas9 等基因编辑技术有可能会修正导致 DMD 的基因异常。另一方面，外显子跳跃疗法旨在透过纠正 RNA 剪接来恢復肌肉营养不良蛋白的产生并减缓肌肉退化。

此外，再生医学方法，包括干细胞疗法和肌肉再生技术，提供了修復和替换受损肌肉组织的潜力。

DMD 治疗的未来前景光明，正在进行的研究和临床研究集中在这些创新疗法，旨在改善治疗效果并提高患者的生活品质。

杜氏肌肉营养不良症 (DMD) 治疗的全球市场动态

影响全球杜氏肌肉营养不良症 (DMD) 治疗市场的关键市场动态是：

主要市场推动因素

DMD 的盛行率和诊断率不断上升：

患有 DMD 的人数不断增加以及诊断技术的进步正在推动治疗需求。根据肌肉萎缩症协会的数据，全球每 3,500 至 5,000 名男性新生儿中，就有 1 名患有 DMD。2014 年《神经流行病学》杂誌发表的一项研究估计，美国 DMD 的盛行率为每 10,000 名 5 至 24 岁男性中有 1.38 人患有。

基因治疗和个人化医疗的进展：

基因治疗和个人化医疗的突破性进展为治疗 DMD 开闢了新的途径。近年来，美国食品药物管理局（FDA）批准了几种针对DMD的基因标靶治疗方法。例如，2016年，FDA批准了Exondys 51（eteplirsen），这是第一个获准用于治疗DMD患者的药物。此后，又有两种外显子跳跃疗法获得批准：2019 年的 Vyondys 53（golodirsen）和 2020 年的 Viltepso（viltolarsen）。

增加研究经费和临床试验：

在研发方面投入了大量资金，并正在进行大量临床试验。根据美国国立卫生研究院 (NIH) RePORTER 资料库显示，到 2020 年，NIH 对杜氏肌肉营养不良症研究的资助将从 2015 年的约 3,300 万美元增加到 8,000 万美元以上。此外，截至 2021 年，ClinicalTrials.gov 列出了 200 多项与 DMD 相关的研究，显示该领域的研究强大。

主要课题

治疗费用高且治疗机会有限：

DMD 治疗，尤其是基因疗法，成本高昂，这可能会降低患者的可及性并限制市场成长。根据 2021 年《Neurology》杂誌发表的一项研究，在美国，治疗 DMD 患者的年度费用在 5 万至 10 万美元之间，具体取决于疾病的阶段。对于新的基因疗法，成本可能会更高。例如，用于治疗脊髓性肌肉萎缩症（另一种神经肌肉疾病）的基因疗法 Zolgensma，每位患者的治疗费用为 100 万美元，是世界上最昂贵的药物之一。

严格的监管要求和冗长的审批流程：

DMD 治疗，尤其是基因疗法，非常复杂，需要漫长而昂贵的临床试验和监管过程。根据塔夫茨大学药物开发研究中心 2018 年的报告，一种罕见疾病治疗（包括 DMD 治疗）从临床试验开始到获得监管部门批准的平均时间为 8.1 年。

病患数量有限和临床试验招募课题：

由于 DMD 是一种罕见疾病，可能难以招募到足够的患者进行临床试验，这可能会减缓研究和开发的速度。根据《孤儿罕见疾病杂誌》的报导，全球每 3,500 至 5,000 名男婴中就有 1 名患有 DMD。这意味着全世界的患者总数估计约为284,000人。

主要趋势

人们对基因疗法和基因编辑方法的关注度日益提高：

基因疗法已成为 DMD 的一种有希望的治疗选择，目前有几种候选疗法正在进行临床试验。根据全球基因疗法临床试验资料库，截至 2021 年，有 20 多项针对 DMD 的基因疗法临床试验正在进行或计划中。美国食品药物管理局 (FDA) 已为几种 DMD 基因治疗方法授予了快速通道和罕见儿科疾病资格，凸显了这种方法日益增长的重要性。

增加合併治疗和多模式治疗方法：

同时针对 DMD 病理多个方面的联合疗法正在得到越来越多的研究。《Journal of Neuromuscular Diseases》上发表的 2020 年一项研究回顾了 53 项 DMD 临床试验。其中，13%正在研究联合疗法，显示多模式治疗的趋势日益增加。

生物标誌物开发和个人化医疗的进展：

开发 DMD 生物标记以实现早期诊断、改善预后和个人化治疗策略的趋势日益明显。美国国立卫生研究院 (NIH) 正在大力投资 DMD 的生物标记研究。根据 NIH RePORTER 资料库，DMD 生物标记研究的资助将从 2015 年的约 500 万美元增加到 2020 年的 1,500 万美元以上。

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目录

第 1 章 全球杜氏肌肉营养不良症 (DMD) 治疗市场简介

• 市场概况
• 调查范围
• 先决条件

第 2 章执行摘要

第3章 VERIFIED MARKET RESEARCH研究方法

• 数据挖掘
• 验证
• 一次资料
• 数据源列表

第 4 章全球杜氏肌肉营养不良症 (DMD) 治疗市场前景

• 概述
• 市场动态
  • 促进因素
  • 阻碍因素
  • 机会
• 波特的五力模型
• 价值链分析

第5章 全球杜氏肌肉营养不良症 (DMD) 治疗市场（依治疗类型）

• 概述
• 类固醇治疗
• 外显子跳跃疗法
• 基因治疗
• 分子治疗

第6章 全球杜氏肌肉营养不良症 (DMD) 治疗市场（依治疗环境划分）

• 概述
• 医院
• 专科诊所
• 家庭医疗

第7章 全球杜氏肌肉营养不良症 (DMD) 治疗市场（按地区）

• 概述
• 北美
  • 美国
  • 加拿大
  • 墨西哥
• 欧洲
  • 德国
  • 英国
  • 法国
  • 欧洲其他地区
• 亚太地区
  • 中国
  • 日本
  • 印度
  • 亚太其他地区
• 世界其他地区
  • 拉丁美洲
  • 中东和非洲

第8章全球杜氏肌肉营养不良症 (DMD) 治疗市场：竞争格局

• 概述
• 各公司市场排名
• 主要发展策略

第9章 公司简介

• Sarepta Therapeutics, Inc
• Santhera Pharmaceuticals
• PTC Therapeutics
• FibroGen, Inc
• ITALFARMACO S.p.A.
• Nobelpharma Co. Ltd
• NS Pharma Inc.
• Pfizer Inc.

第10章 主要进展

• 产品发布/开发
• 併购
• 业务拓展
• 伙伴关係和联盟

第11章 附录

• 相关研究

Duchenne Muscular Dystrophy (DMD) Treatment Market Size And Forecast

The Duchenne Muscular Dystrophy (DMD) Treatment Market size was valued at USD 722.79 Million in 2024 and is projected to reach USD 1905.24 Million by 2031 , growing at a CAGR of 12.88% from 2024 to 2031. Duchenne muscular dystrophy (DMD) treatment primarily aims to manage symptoms, slow disease progression, and enhance the quality of life, as there is currently no cure for this condition. Corticosteroids like prednisone and deflazacort are commonly prescribed to help maintain muscle strength and function, despite potential side effects. Physical therapy and tailored exercise programs play a crucial role in preserving mobility and preventing contractures.

In addition to corticosteroids, medications such as idebenone and eteplirsen may address specific challenges associated with DMD. Promising emerging therapies, including gene therapy and exon-skipping medications, target the genetic basis of DMD and could potentially lead to long-term improvements.

Gene editing techniques, such as CRISPR-Cas9, hold the potential to correct the genetic abnormalities responsible for DMD while exon-skipping therapies aim to restore dystrophin production by modifying RNA splicing, thereby slowing muscle degeneration.

Furthermore, regenerative medicine approaches that involve stem cell therapies and muscle regeneration techniques offer the possibility of healing and replacing damaged muscle tissue.

The future of DMD treatment looks promising, with ongoing research and clinical studies focused on these innovative therapies, aiming to enhance patient outcomes and improve their quality of life.

Global Duchenne Muscular Dystrophy (DMD) Treatment Market Dynamics

The key market dynamics that are shaping the global duchenne muscular dystrophy (DMD) treatment market include:

Key Market Drivers

Increasing Prevalence and Diagnosis Of DMD:

The rising number of DMD cases, coupled with improved diagnostic techniques, is driving the demand for treatments. According to the Muscular Dystrophy Association, DMD affects approximately 1 in every 3,500 to 5,000 male births worldwide. A study published in the journal Neuroepidemiology in 2014 estimated the prevalence of DMD in the United States to be 1.38 per 10,000 males aged 5-24 years.

Advancements in Gene Therapy and Personalized Medicine:

Breakthroughs in gene therapy and personalized treatment approaches are opening new avenues for DMD treatment. The U.S. Food and Drug Administration (FDA) has approved several gene-targeted therapies for DMD in recent years. For instance, in 2016, the FDA approved Exondys 51 (eteplirsen), the first drug approved to treat patients with DMD. Since then, two more exon-skipping therapies have been approved: Vyondys 53 (golodirsen) in 2019 and Viltepso (viltolarsen) in 2020.

Increased Research Funding and Clinical Trials:

Substantial investments in research and development, along with numerous ongoing clinical trials. According to the National Institutes of Health (NIH) RePORTER database, NIH funding for Duchenne Muscular Dystrophy research increased from approximately $33 million in 2015 to over $80 million in 2020. Additionally, as of 2021, ClinicalTrials.gov listed over 200 studies related to DMD, indicating significant ongoing research efforts in this field.

Key Challenges

High Cost of Treatment and Limited Accessibility:

The expensive nature of DMD treatments, particularly gene therapies, can limit market growth by reducing accessibility for patients. According to a study published in the journal Neurology in 2021, the annual cost of care for patients with DMD in the United States ranges from $50,000 to $100,000, depending on the stage of the disease. For newer gene therapies, the costs can be even higher. For instance, Zolgensma, a gene therapy for spinal muscular atrophy (another neuromuscular disorder), is priced at USD1 Million per patient, making it one of the world's most expensive drugs.

Stringent Regulatory Requirements and Long Approval Processes:

The complex nature of DMD treatments, especially gene therapies, leads to lengthy and costly clinical trials and regulatory processes. A report by the Tufts Center for the Study of Drug Development in 2018 found that the average time from the start of clinical testing to regulatory approval for rare disease drugs, including those for DMD, is 8.1 years.

Limited Patient Population and Challenges in Clinical Trial Recruitment:

The rarity of DMD makes it difficult to recruit sufficient patients for clinical trials, potentially slowing down research and development efforts. According to the Orphanet Journal of Rare Diseases, DMD affects approximately 1 in 3,500 to 5,000 male births worldwide. This translates to an estimated patient population of about 284,000 individuals globally.

Key Trends

Increasing Focus on Gene Therapy and Gene Editing Approaches:

Gene therapy is emerging as a promising treatment option for DMD, with several candidates in clinical trials. According to the Gene Therapy Clinical Trials Worldwide database, as of 2021, there were over 20 ongoing or planned gene therapy clinical trials for DMD. The U.S. Food and Drug Administration (FDA) has granted Fast Track and Rare Pediatric Disease designations to multiple gene therapy candidates for DMD, highlighting the growing importance of this approach.

Rise In Combination Therapies and Multi-Modal Treatment Approaches:

Researchers are increasingly exploring combination therapies that target multiple aspects of DMD pathology simultaneously. A study published in the Journal of Neuromuscular Diseases in 2020 reviewed 53 clinical trials for DMD. Of these, 13% were investigating combination therapies, indicating a growing trend towards multi-modal treatment approaches.

Advancement in Biomarker Development and Personalized Medicine:

There's a growing trend towards developing biomarkers for DMD to enable earlier diagnosis, better prognosis, and personalized treatment strategies. The National Institutes of Health (NIH) has invested significantly in biomarker research for DMD. According to the NIH RePORTER database, funding for DMD biomarker research increased from approximately USD 5 Million in 2015 to over $15 million in 2020.

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Global Duchenne Muscular Dystrophy (DMD) Treatment Market Regional Analysis

Here is a more detailed regional analysis of the global duchenne muscular dystrophy (DMD) treatment market:

North America

North America substantially dominates the global Duchenne Muscular Dystrophy treatment market owing to the high prevalence of DMD.

The relatively high incidence rate of DMD in North America contributes to the region's market dominance. According to the Centers for Disease Control and Prevention (CDC), DMD affects approximately 1 in every 3,500 to 5,000 male births in the United States.

Substantial funding for DMD research has accelerated the development of new treatments. The National Institutes of Health (NIH) reported awarding over $76 million in research grants for muscular dystrophy in fiscal year 2020, with a significant portion dedicated to DMD research.

The U.S. Food and Drug Administration (FDA) has implemented policies to expedite the approval process for DMD treatments. As of 2023, the FDA has approved four exon-skipping therapies for DMD, demonstrating the impact of these regulatory initiatives on bringing new treatments to market.

In North America, healthcare reimbursement policies and insurance coverage significantly enhance the accessibility of Duchenne muscular dystrophy (DMD) treatments for eligible patients.

Government-funded healthcare programs, private insurance plans, and patient assistance initiatives collectively help alleviate the financial burden associated with DMD treatment costs for patients and their families.

This favorable reimbursement landscape incentivizes pharmaceutical companies to invest in the research, development, and commercialization of DMD therapies, facilitating broader access to life-changing treatments.

Asia Pacific

Asia Pacific is anticipated to witness the fastest growth in the global Duchenne Muscular Dystrophy treatment market during the forecast period owing to the large patient population and increasing diagnosis rates.

The Asia Pacific region has a significant DMD patient population due to its large overall population. In India alone, it's estimated that there are about 5 out of every 100,000 male births affected by DMD, according to a study published in the Annals of the Indian Academy of Neurology.

Improved diagnostic capabilities in countries like China and Japan have led to earlier and more accurate DMD diagnoses.

Economic growth in many Asia Pacific countries has led to increased healthcare spending. For instance, China's healthcare expenditure grew from 5.03% of GDP in 2010 to 6.57% in 2020, according to World Bank data. This growth has supported the development of specialized care facilities for rare diseases like DMD.

Expanding clinical research and treatment options There's been a significant increase in DMD-related clinical trials in the Asia Pacific region. According to the U.S. National Library of Medicine's ClinicalTrials.gov database, as of 2023, there were over 50 ongoing or completed clinical trials for DMD treatments in countries like China, Japan, and South Korea combined. This surge in research activity is driving innovation and expanding treatment options for DMD patients in the region.

Global Duchenne Muscular Dystrophy (DMD) Treatment Market: Segmentation Analysis

The Global Duchenne Muscular Dystrophy (DMD) Treatment Market is segmented based on Treatment Type, Treatment Setting, And Geography.

Duchenne Muscular Dystrophy (DMD) Treatment Market, By Treatment Type

• Steroid Therapy0
• Exon Skipping Therapy
• Gene Therapy
• Molecular-based Therapy

Based on the Treatment Type, the Global Duchenne Muscular Dystrophy Treatment Market is bifurcated into Steroid Therapy, Exon Skipping Therapy, Gene Therapy, and Molecular-based Therapy. Steroid therapy leads the Global Duchenne Muscular Dystrophy (DMD) Treatment Market, primarily due to its widespread use and established effectiveness in slowing disease progression. Corticosteroids, such as prednisone and deflazacort, are recognized as the standard of care for DMD, helping to preserve muscle function and delay the progression of the disease.

Duchenne Muscular Dystrophy (DMD) Treatment Market, By Treatment Setting

• Hospitals
• Specialty Clinics
• Homecare

Based on the Treatment Setting, the Global Duchenne Muscular Dystrophy (DMD) Treatment Market is bifurcated into Hospitals, Specialty Clinics, and Homecare. The Hospital segment dominates the Global Duchenne Muscular Dystrophy (DMD) Treatment Market owing to its ability to provide comprehensive care that encompasses diagnosis, treatment, rehabilitation, and support services. Hospitals typically have access to advanced therapies, including gene therapy and stem cell therapy, which are essential for managing DMD effectively.

Duchenne Muscular Dystrophy (DMD) Treatment Market, By Geography

• North America
• Europe
• Asia Pacific
• Rest of the World

Based on Geography, the Global Duchenne Muscular Dystrophy (DMD) Treatment Market is classified into North America, Europe, Asia Pacific, and the Rest of the World. North America substantially dominates the global Duchenne Muscular Dystrophy (DMD) treatment market owing to the high prevalence of DMD. The relatively high incidence rate of DMD in North America contributes to the region's market dominance. According to the Centers for Disease Control and Prevention (CDC), DMD affects approximately 1 in every 3,500 to 5,000 male births in the United States. Substantial funding for DMD research has accelerated the development of new treatments. The National Institutes of Health (NIH) reported awarding over $76 million in research grants for muscular dystrophy in fiscal year 2020, with a significant portion dedicated to DMD research.

Key Players

• The "Global Duchenne Muscular Dystrophy (DMD) Treatment Market" study report will provide valuable insight with an emphasis on the global market. The major players in the market are
• Sarepta Therapeutics, Inc., Santhera Pharmaceuticals, PTC Therapeutics, FibroGen, Inc., ITALFARMACO S.p.A., Nobelpharma Co. Ltd, NS Pharma, Inc., Pfizer Inc.
• This section offers in-depth analysis through a company overview, position analysis, the regional and industrial footprint of the company, and the ACE matrix for insightful competitive analysis. The section also provides an exhaustive analysis of the financial performances of mentioned players in the given market.

Our market analysis also entails a section solely dedicated to such major players wherein our analysts provide an insight into the financial statements of all the major players, along with product benchmarking and SWOT analysis. The competitive landscape section also includes key development strategies, market share, and market ranking analysis of the above-mentioned players globally.

Global Duchenne Muscular Dystrophy (DMD) Treatment Market Key Developments

• In June 2023, Sarepta Therapeutics received accelerated FDA approval for ELEVIDYS(TM) (delandistrogene moxeparvovec-rokl), the first gene therapy for DMD. This ground-breaking medication has been approved for ambulatory patients aged 4-5 years who have a verified DMD mutation.
• In September 2023, PTC Therapeutics reported positive findings from their Phase 3 MOVE study of vatiquinone for mitochondrial dysfunction and DMD-related fatigue. The company intends to submit a New Drug Application to the FDA within the first half of 2024.
• In December 2023, Solid Biosciences announced the collaboration with AavantiBio to build a new firm focused on developing genetic therapies for rare diseases such as DMD. The United company wants to speed up the development of gene treatments for muscular dystrophy.
• In February 2024, Pfizer discontinued its Phase 3 research of fordadistrogene movaparvovec, a miniature dystrophin gene DMD therapy is not recommended due to safety concerns. These rulings change the competitive landscape for gene treatments in DMD treatment.
• In April 2024, Edgewise Therapeutics begins a Phase 2 trial for EDG-5506, a revolutionary muscle-preserving DMD treatment. The medicine is intended to protect skeletal and heart muscle in DMD patients, potentially reducing disease development.

TABLE OF CONTENTS

1 INTRODUCTION OF GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET

• 1.1 Overview of the Market
• 1.2 Scope of Report
• 1.3 Assumptions

2 EXECUTIVE SUMMARY

3 RESEARCH METHODOLOGY OF VERIFIED MARKET RESEARCH

• 3.1 Data Mining
• 3.2 Validation
• 3.3 Primary Interviews
• 3.4 List of Data Sources

4 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET OUTLOOK

• 4.1 Overview
• 4.2 Market Dynamics
  • 4.2.1 Drivers
  • 4.2.2 Restraints
  • 4.2.3 Opportunities
• 4.3 Porters Five Force Model
• 4.4 Value Chain Analysis

5 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY TREATMENT TYPE

• 5.1 Overview
• 5.2 Steroid Therapy
• 5.3 Exon Skipping Therapy
• 5.4 Gene Therapy
• 5.5 Molecular-based Therapy

6 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY TREATMENT SETTING

• 6.1 Overview
• 6.2 Hospitals
• 6.3 Specialty Clinics
• 6.4 Homecare

7 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET, BY GEOGRAPHY

• 7.1 Overview
• 7.2 North America
  • 7.2.1 U.S.
  • 7.2.2 Canada
  • 7.2.3 Mexico
• 7.3 Europe
  • 7.3.1 Germany
  • 7.3.2 U.K.
  • 7.3.3 France
  • 7.3.4 Rest of Europe
• 7.4 Asia Pacific
  • 7.4.1 China
  • 7.4.2 Japan
  • 7.4.3 India
  • 7.4.4 Rest of Asia Pacific
• 7.5 Rest of the World
  • 7.5.1 Latin America
  • 7.5.2 Middle East And Africa

8 GLOBAL DUCHENNE MUSCULAR DYSTROPHY TREATMENT MARKET COMPETITIVE LANDSCAPE

• 8.1 Overview
• 8.2 Company Market Ranking
• 8.3 Key Development Strategies

9 COMPANY PROFILES

• 9.1 Sarepta Therapeutics, Inc
  • 9.1.1 Overview
  • 9.1.2 Financial Performance
  • 9.1.3 Product Outlook
  • 9.1.4 Key Developments
• 9.2 Santhera Pharmaceuticals
  • 9.2.1 Overview
  • 9.2.2 Financial Performance
  • 9.2.3 Product Outlook
  • 9.2.4 Key Developments
• 9.3 PTC Therapeutics
  • 9.3.1 Overview
  • 9.3.2 Financial Performance
  • 9.3.3 Product Outlook
  • 9.3.4 Key Developments
• 9.4 FibroGen, Inc
  • 9.4.1 Overview
  • 9.4.2 Financial Performance
  • 9.4.3 Product Outlook
  • 9.4.4 Key Developments
• 9.5 ITALFARMACO S.p.A.
  • 9.5.1 Overview
  • 9.5.2 Financial Performance
  • 9.5.3 Product Outlook
  • 9.5.4 Key Developments
• 9.6 Nobelpharma Co. Ltd
  • 9.6.1 Overview
  • 9.6.2 Financial Performance
  • 9.6.3 Product Outlook
  • 9.6.4 Key Developments
• 9.7 NS Pharma Inc.
  • 9.7.1 Overview
  • 9.7.2 Financial Performance
  • 9.7.3 Product Outlook
  • 9.7.4 Key Developments
• 9.8 Pfizer Inc.
  • 9.8.1 Overview
  • 9.8.2 Financial Performance
  • 9.8.3 Product Outlook
  • 9.8.4 Key Developments

10 KEY DEVELOPMENTS

• 10.1 Product Launches/Developments
• 10.2 Mergers and Acquisitions
• 10.3 Business Expansions
• 10.4 Partnerships and Collaborations

11 Appendix

• 11.1 Related Research