查看購物車
  • English
  • Japanese
  • Korean
封面
市場調查報告書
商品編碼
2071554

裘馨氏肌肉失養症（DMD）：市場展望、流行病學、競爭格局、市場預測報告（2025-2035年）

Duchenne Muscular Dystrophy (DMD) - Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report - 2025 To 2035
出版日期: | 出版商: Thelansis Knowledge Partners | 英文 154 Pages | 商品交期: 2-3個工作天內

價格
簡介目錄

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 連鎖隱性遺傳性神經肌肉疾病，由 DMD 基因突變引起，導致Dystrophin的生成受阻。這會導致骨骼肌、心肌和呼吸肌肉的筋膜進行性無力、慢性發炎以及纖維脂質替代。男孩在嬰兒期會出現近端肌無力、戈爾氏徵、小腿假性肥大和運動發育遲緩，並在青春期早期發展至喪失行走能力，此後呼吸衰竭和心肌病成為主要死因。診斷需要結合肌酸激酶水平顯著升高、Dystrophin免疫組化染色和 DMD 基因定序才能確診。治療的基礎仍然是糖皮質激素，其中地夫可特和伐洛龍的耐受性有所不同。諸如eteplirsen、golodyrsen、virtolarsen和casimersen等外顯子跳躍反義寡核苷酸可部分恢復合格患者的Dystrophin。 Delandistrogen moxeparbovec是腺結合病毒（AAV）的基因療法，可將功能性截短Dystrophin遞送至廣泛的合格條件的患者。 Gibinostat是First-in-Class蛋白去乙醯化酶（HDAC）抑制劑，已獲準用於6歲及以上患者，它直接靶向下游纖維脂質肌肉退化，並可作為肌營養不良Dystrophin恢復策略的補充。心臟監測、非侵入性通氣支持、物理治療和整形外科治療至關重要。現代多學科方法已顯著改善預後。遺傳諮詢、心理支持和以家庭為中心的教育對於富有同情心的長期管理至關重要。

主要亮點

  • 據預測，德國患有 DMD 的人數將從 2025 年的 2171 人增加到 2035 年的 2193 人。
  • 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。
  • 基因療法和突變標靶療法正在改變治療疾病的方式。

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 G8 市場（美國、歐盟 5 國、日本、中國）的藥物開發與生命週期管理策略？
  • 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

  • G8
    • 美國
    • EU5
      • 法國
      • 德國
      • 義大利
      • 西班牙
      • 英國
    • 日本
    • 中國

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：主要調查結果及分析師說明

  • 主要趨勢：市場概況、SWOT分析、商業利益與風險等。

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
  • 值得關注的早期研發管線

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

簡介目錄

Duchenne Muscular Dystrophy (DMD) Market Outlook

Thelansis's "Duchenne Muscular Dystrophy (DMD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report - 2025 To 2035" covers disease overview, epidemiology, 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 eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Duchenne Muscular Dystrophy (DMD) Overview

Duchenne muscular dystrophy (DMD) is a severe X-linked recessive neuromuscular disorder caused by DMD gene mutations abolishing dystrophin production, triggering progressive muscle membrane fragility, chronic inflammation, and fibrofatty replacement of skeletal, cardiac, and respiratory musculature. Boys present in early childhood with proximal weakness, Gowers sign, calf pseudohypertrophy, and delayed motor milestones, progressing to loss of ambulation by early adolescence, followed by respiratory insufficiency and cardiomyopathy as principal causes of mortality. Diagnosis integrates markedly elevated creatine kinase, dystrophin immunostaining, and confirmatory DMD genetic sequencing. Corticosteroids remain the therapeutic cornerstone, with deflazacort and vamorolone offering differing tolerability profiles. Exon-skipping antisense oligonucleotides including eteplirsen, golodirsen, viltolarsen, and casimersen restore partial dystrophin in mutation-eligible patients. Delandistrogene moxeparvovec, an AAV-based gene therapy, delivers truncated functional dystrophin broadly across eligible patients. Givinostat, a first-in-class HDAC inhibitor approved for patients aged six and older, directly targets downstream fibrofatty muscle transformation, complementing dystrophin-restorative strategies. Cardiac surveillance, non-invasive ventilatory support, physiotherapy, and orthopaedic management are essential. Prognosis has improved considerably with modern multidisciplinary approaches; genetic counselling, psychological support, and family-centred education are integral to compassionate long-term management.

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.

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 G8 markets (US, EU5, Japan, and China)?
  • 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

  • G8
    • United States
    • EU5
      • France
      • Germany
      • Italy
      • Spain
      • U.K.
    • Japan
    • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

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)