裘馨氏肌肉失養症（DMD）：全球市場展望、流行病學、競爭格局和市場預測報告（32個主要市場），2025-2035年

市場概覽

• 德國DMD市場預計將從2025年的2.94億美元成長到2035年的8.19億美元。
• 以下因素正在推動市場成長：
• 昂貴的基因療法和外顯子跳躍療法的普及
• 擴大可治療疾病範圍並改善診斷技術。
• 預計未來市場規模將受到下一代基因療法的極大影響。

裘馨氏肌肉失養症（DMD）概述

裘馨氏肌肉失養症(DMD) 是嚴重的X 連鎖隱性遺傳疾病，由 DMD 基因突變引起，導致Dystrophin生成受阻，進而引起筋膜脆弱、慢性發炎和纖維脂質性肌肉變性。男性患者表現為近端肌無力、戈爾氏徵和小腿假性肥大，病情進展至活動受限、呼吸衰竭和致命性心肌病。診斷需要肌酸激酶水平升高、免疫組織化學或基因定序。地夫可特和伐洛龍等糖皮質激素仍是標準治療方法。標靶治療包括針對特定突變的外顯子跳躍反義寡核苷酸和基於腺相關病毒 (AAV) 的基因療法，例如德蘭氏莫西帕博維克。截至 2026 年，已通過核准的組蛋白去乙醯化酶 (HDAC) 抑制劑吉比司他可直接抑制下游纖維脂質性肌變性，目前也用於 6 個月及以上年齡的患者。主動心臟監測、非侵入性通氣和物理治療是長期照護的重要組成部分。

主要亮點

• 預計德國的 DMD 病例數將從 2025 年的 2171 例增加到 2035 年的 2193 例。
• DMD 是一種罕見的進行性神經肌肉疾病，由Dystrophin基因突變引起，導致不可逆的肌肉退化。
• 支持性照護的改善提高了存活率，導致成年 DMD 患者數量增加。
• 基因療法和針對突變的療法正在改變我們治療疾病的方式。

市場定義：

• 北美洲（美國、加拿大）
• 歐洲（奧地利、比利時、捷克、丹麥、芬蘭、法國、德國、希臘、義大利、荷蘭、挪威、波蘭、葡萄牙、俄羅斯、西班牙、瑞典、瑞士、英國）
• 中東（沙烏地阿拉伯、阿拉伯聯合大公國、科威特、土耳其）
• 亞太地區（澳洲、中國、香港、印度、印尼、日本、馬來西亞、紐西蘭、菲律賓、新加坡、韓國、台灣、泰國、越南）
• 非洲（埃及、奈及利亞、南非、摩洛哥）
• 南美洲/拉丁美洲（阿根廷、巴西、智利、哥倫比亞、墨西哥、秘魯）

格式化和更新訊息

• 詳細報告（PDF）
• 市場預測模型（基於微軟Excel）
• 流行病學數據（MS Excel，互動式工具）
• 高階主管洞察（PPT簡報）
• 其他功能：定期更新、自訂和顧問支援。
• 根據 Thelansis 的政策，我們確保所有最新更新在發布前都反映在報告內容和市場模型中。

主要問題

• 如何最佳化在 32 個市場（北美、歐洲、中東、亞太、非洲和南美/拉丁美洲）的藥物開發和生命週期管理策略？
• 從發病率、盛行率、人群組成以及接受藥物治療的患者人數來看，患者數量分別是多少？
• 未來十年市場收入和病患佔有率的預測是多少？
• 哪些因素對市場趨勢影響最大？
• 受訪專家對目前和新興的治療方法有何看法？
• 哪款在研發線產品最有前景？其上市潛力及未來市場定位如何？
• 主要未被滿足的需求是什麼？ KOL 對目標受眾有何期望？
• 為確保藥物獲得批准並順利進入市場，必須滿足哪些關鍵的監管和支付方要求？

目標國家

• 北美洲（美國、加拿大）
• 歐洲（奧地利、比利時、捷克、丹麥、芬蘭、法國、德國、希臘、義大利、荷蘭、挪威、波蘭、葡萄牙、俄羅斯、西班牙、瑞典、瑞士、英國）
• 中東（沙烏地阿拉伯、阿拉伯聯合大公國、科威特、土耳其）
• 亞太地區（澳洲、中國、香港、印度、印尼、日本、馬來西亞、紐西蘭、菲律賓、新加坡、韓國、台灣、泰國、越南）
• 非洲（埃及、奈及利亞、南非、摩洛哥）
• 南美洲/拉丁美洲（阿根廷、巴西、智利、哥倫比亞、墨西哥、秘魯）

大公司

• Solid Biosciences Inc.
• Sarepta Therapeutics, Inc.
• REGENXBIO Inc.
• Edgewise Therapeutics, Inc.
• Santhera Pharmaceuticals
• Daiichi Sankyo Co., Ltd.
• Pfizer
• Hoffmann-La Roche
• Insmed Gene Therapy LLC
• BioMarin Pharmaceutical
• Taiho Pharmaceutical Co., Ltd.
• Capricor Inc.
• NS Pharma, Inc.
• PTC Therapeutics
• Entrada Therapeutics, Inc.
• Satellos Bioscience, Inc.
• Dyne Therapeutics
• Nippon Shinyaku Co., Ltd.
• Avidity Biosciences, Inc.
• Cumberland Pharmaceuticals
• Precision BioSciences, Inc.
• Belief BioMed（Beijing）Co., Ltd.

目錄

第1章：主要調查結果和分析師說明

• 主要趨勢：市場概況、SWOT分析、商業性利益與風險等。

第2章：疾病背景

• 疾病定義、分類、病因和病理生理學、藥物標靶等。

第3章：流行病學

• 重點
• 發病率/盛行率
• 已確診並正在接受藥物治療的患者族群。
• 合併症
• 其他相關患者群

第4章 市場規模及預測

• 重點
• 市場促進因素與限制因素
• 按藥物類別分類的趨勢
• 各國具體趨勢

第5章 競爭情勢

• 目前的治療方法
• 重點
• 診斷和治療過程/演算法
• 目前主要療法－概述及關鍵意見領袖見解
• 新興療法
• 重點
• 值得關注的後期新興療法—概述、市場上市預期和關鍵意見領袖見解
• 值得關注的早期管道

第6章：未滿足的需求與目標產品分析

• 尚未滿足的主要需求及其透過新興療法實現的潛力。
• TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

Global Duchenne Muscular Dystrophy (DMD) Market Outlook

Thelansis's "Global Duchenne Muscular Dystrophy (DMD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report (Top 32 Markets) - 2025 To 2035" covers disease overview, epidemiology, kidney biopsy cases, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Duchenne Muscular Dystrophy treatment modalities options for the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America).

Duchenne Muscular Dystrophy (DMD) Overview

Duchenne muscular dystrophy (DMD) is a severe X-linked recessive disorder caused by DMD gene mutations abolishing dystrophin production, causing muscle membrane fragility, chronic inflammation, and fibrofatty muscle degeneration. Boys present with proximal weakness, Gowers sign, and calf pseudohypertrophy, progressing to loss of ambulation, respiratory insufficiency, and fatal cardiomyopathy. Diagnosis requires elevated creatine kinase, immunostaining, or genetic sequencing. Corticosteroids like deflazacort and vamorolone remain foundational. Targeted therapies include exon-skipping antisense oligonucleotides for specific mutations and the AAV-based gene therapy delandistrogene moxeparvovec. Management in 2026 also utilizes the approved HDAC inhibitor givinostat for patients aged six months and older to directly mitigate downstream fibrofatty muscle transformation. Proactive cardiac surveillance, non-invasive ventilation, and physical therapy are mandatory components of long-term care.

Key Highlights

• In Germany, prevalent DMD cases are projected to increase from 2,171 in 2025 to 2,193 by 2035.
• DMD is a rare, progressive neuromuscular disorder caused by dystrophin gene mutations, leading to irreversible muscle degeneration.
• Improved supportive care has increased survival, resulting in a larger adult DMD population.
• Gene therapies and mutation-targeted treatments are transforming the treatment landscape.

Market Overview

• The Germany DMD market is projected to grow from $294M in 2025 to $819M by 2035.
• Market growth is driven by:
• Uptake of high-cost gene and exon-skipping therapies
• Expanded treatment eligibility and improved diagnosis
• Future market value will be heavily influenced by next-generation genetic therapies.

Market Definition:

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

Insights driven by robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support
• As per Thelansis's policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2025-2035)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market's trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?

Countries Covered

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

Companies Mentioned

• Solid Biosciences Inc.
• Sarepta Therapeutics, Inc.
• REGENXBIO Inc.
• Edgewise Therapeutics, Inc.
• Santhera Pharmaceuticals
• Daiichi Sankyo Co., Ltd.
• Pfizer
• Hoffmann-La Roche
• Insmed Gene Therapy LLC
• BioMarin Pharmaceutical
• Taiho Pharmaceutical Co., Ltd.
• Capricor Inc.
• NS Pharma, Inc.
• PTC Therapeutics
• Entrada Therapeutics, Inc.
• Satellos Bioscience, Inc.
• Dyne Therapeutics
• Nippon Shinyaku Co., Ltd.
• Avidity Biosciences, Inc.
• Cumberland Pharmaceuticals
• Precision BioSciences, Inc.
• Belief BioMed (Beijing) Co., Ltd.

Table of Contents

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risks, etc.

2. Disease Context

• Disease definition, classification, etiology and pathophysiology, drug targets, etc.

3. Epidemiology

• Key takeaways
• Incidence / Prevalence
• Diagnosed and Drug-Treated populations
• Comorbidities
• Other relevant patient segments

4. Market Size and Forecast

• Key takeaways
• Market drivers and constraints
• Drug-class specific trends
• Country-specific trends

5. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Notable late-phase emerging therapies - profiles, launch expectations, KOL insights
• Notable early-phase pipeline

6. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

7. Regulatory and Reimbursement Environments (by country and payer insights)

8. Appendix (e.g., bibliography, methodology)