全球杜氏肌肉營養不良症 (DMD) 治療市場規模（按治療類型、治療環境、地區、範圍和預測）

杜氏肌肉營養不良症 (DMD) 治療市場規模及預測

杜氏肌肉營養不良症 (DMD) 治療市場規模在 2024 年價值 7.2279 億美元，預計到 2031 年將達到 19.0524 億美元，2024 年至 2031 年的複合年增長率為 12.88%。杜氏肌肉營養不良症 (DMD) 治療的主要目標是控制症狀、減緩病情進展和改善生活品質。儘管有潛在的副作用，但通常使用潑尼鬆或地夫可特等皮質類固醇來幫助維持肌肉力量和功能。物理治療和個人化的運動計畫在維持活動能力和預防攣縮方面發揮重要作用。

除了皮質類固醇外，艾地苯醌和依替普利生等藥物也許能夠解決與 DMD 相關的某些課題。基因療法和外顯子跳躍藥物等有前景的新療法針對的是 DMD 的遺傳基礎，並可能帶來長期改善。

CRISPR-Cas9 等基因編輯技術有可能會修正導致 DMD 的基因異常。另一方面，外顯子跳躍療法旨在透過糾正 RNA 剪接來恢復肌肉營養不良蛋白的產生並減緩肌肉退化。

此外，再生醫學方法，包括幹細胞療法和肌肉再生技術，提供了修復和替換受損肌肉組織的潛力。

DMD 治療的未來前景光明，正在進行的研究和臨床研究集中在這些創新療法，旨在改善治療效果並提高患者的生活品質。

杜氏肌肉營養不良症 (DMD) 治療的全球市場動態

影響全球杜氏肌肉營養不良症 (DMD) 治療市場的關鍵市場動態是：

主要市場推動因素

DMD 的盛行率和診斷率不斷上升：

患有 DMD 的人數不斷增加以及診斷技術的進步正在推動治療需求。根據肌肉萎縮症協會的數據，全球每 3,500 至 5,000 名男性新生兒中，就有 1 名患有 DMD。2014 年《神經流行病學》雜誌發表的一項研究估計，美國 DMD 的盛行率為每 10,000 名 5 至 24 歲男性中有 1.38 人患有。

基因治療和個人化醫療的進展：

基因治療和個人化醫療的突破性進展為治療 DMD 開闢了新的途徑。近年來，美國食品藥物管理局（FDA）批准了幾種針對DMD的基因標靶治療方法。例如，2016年，FDA批准了Exondys 51（eteplirsen），這是第一個獲準用於治療DMD患者的藥物。此後，又有兩種外顯子跳躍療法獲得批准：2019 年的 Vyondys 53（golodirsen）和 2020 年的 Viltepso（viltolarsen）。

增加研究經費和臨床試驗：

在研發方面投入了大量資金，並正在進行大量臨床試驗。根據美國國立衛生研究院 (NIH) RePORTER 資料庫顯示，到 2020 年，NIH 對杜氏肌肉營養不良症研究的資助將從 2015 年的約 3,300 萬美元增加到 8,000 萬美元以上。此外，截至 2021 年，ClinicalTrials.gov 列出了 200 多項與 DMD 相關的研究，顯示該領域的研究強大。

主要課題

治療費用高且治療機會有限：

DMD 治療，尤其是基因療法，成本高昂，這可能會降低患者的可及性並限制市場成長。根據 2021 年《Neurology》雜誌發表的一項研究，在美國，治療 DMD 患者的年度費用在 5 萬至 10 萬美元之間，具體取決於疾病的階段。對於新的基因療法，成本可能會更高。例如，用於治療脊髓性肌肉萎縮症（另一種神經肌肉疾病）的基因療法 Zolgensma，每位患者的治療費用為 100 萬美元，是世界上最昂貴的藥物之一。

嚴格的監管要求和冗長的審批流程：

DMD 治療，尤其是基因療法，非常複雜，需要漫長而昂貴的臨床試驗和監管過程。根據塔夫茨大學藥物開發研究中心 2018 年的報告，一種罕見疾病治療（包括 DMD 治療）從臨床試驗開始到獲得監管部門批准的平均時間為 8.1 年。

病患數量有限和臨床試驗招募課題：

由於 DMD 是一種罕見疾病，可能難以招募到足夠的患者進行臨床試驗，這可能會減緩研究和開發的速度。根據《孤兒罕見疾病雜誌》的報導，全球每 3,500 至 5,000 名男嬰中就有 1 名患有 DMD。這意味著全世界的患者總數估計約為284,000人。

主要趨勢

人們對基因療法和基因編輯方法的關注度日益提高：

基因療法已成為 DMD 的一種有希望的治療選擇，目前有幾種候選療法正在進行臨床試驗。根據全球基因療法臨床試驗資料庫，截至 2021 年，有 20 多項針對 DMD 的基因療法臨床試驗正在進行或計劃中。美國食品藥物管理局 (FDA) 已為幾種 DMD 基因治療方法授予了快速通道和罕見兒科疾病資格，凸顯了這種方法日益增長的重要性。

增加合併治療和多模式治療方法：

同時針對 DMD 病理多個方面的聯合療法正在得到越來越多的研究。《Journal of Neuromuscular Diseases》上發表的 2020 年一項研究回顧了 53 項 DMD 臨床試驗。其中，13%正在研究聯合療法，顯示多模式治療的趨勢日益增加。

生物標誌物開發和個人化醫療的進展：

開發 DMD 生物標記以實現早期診斷、改善預後和個人化治療策略的趨勢日益明顯。美國國立衛生研究院 (NIH) 正在大力投資 DMD 的生物標記研究。根據 NIH RePORTER 資料庫，DMD 生物標記研究的資助將從 2015 年的約 500 萬美元增加到 2020 年的 1,500 萬美元以上。

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目錄

第 1 章 全球杜氏肌肉營養不良症 (DMD) 治療市場簡介

• 市場概況
• 調查範圍
• 先決條件

第 2 章執行摘要

第3章 VERIFIED MARKET RESEARCH研究方法

• 數據挖掘
• 驗證
• 一次資料
• 數據源列表

第 4 章全球杜氏肌肉營養不良症 (DMD) 治療市場前景

• 概述
• 市場動態
  • 促進因素
  • 阻礙因素
  • 機會
• 波特的五力模型
• 價值鏈分析

第5章 全球杜氏肌肉營養不良症 (DMD) 治療市場（依治療類型）

• 概述
• 類固醇治療
• 外顯子跳躍療法
• 基因治療
• 分子治療

第6章 全球杜氏肌肉營養不良症 (DMD) 治療市場（依治療環境劃分）

• 概述
• 醫院
• 專科診所
• 家庭醫療

第7章 全球杜氏肌肉營養不良症 (DMD) 治療市場（按地區）

• 概述
• 北美
  • 美國
  • 加拿大
  • 墨西哥
• 歐洲
  • 德國
  • 英國
  • 法國
  • 歐洲其他地區
• 亞太地區
  • 中國
  • 日本
  • 印度
  • 亞太其他地區
• 世界其他地區
  • 拉丁美洲
  • 中東和非洲

第8章全球杜氏肌肉營養不良症 (DMD) 治療市場：競爭格局

• 概述
• 各公司市場排名
• 主要發展策略

第9章 公司簡介

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

第10章 主要進展

• 產品發佈/開發
• 併購
• 業務拓展
• 夥伴關係和聯盟

第11章 附錄

• 相關研究

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