1 型強直性肌肉營養不良症 (DM1)：市場展望、流行病學、競爭格局、市場預測報告（2025-2035 年）

市場概覽

• 儘管德國 DM1 患者數量相對較少，但預計到 2035 年，德國 DM1 市場將從 2025 年的約 7,800 萬美元成長到 2035 年的約 2.45 億美元，呈現兩位數的複合年成長率（約 12-13%），這主要得益於研發管線治療方法和新療法的出現。
• 患者數量僅略有增加（年複合成長率約為 0.4%），這表明該疾病的流行情況穩定，發病率的變化對市場擴張的影響有限。

1 型強直性肌肉營養不良症 (DM1) 概述

1型強直性肌肉營養不良症（DM1）是一種多系統性常染色體顯性遺傳神經肌肉疾病，由DMPK基因（19號染色體，q13.3）3'非翻譯區中CTG三核苷酸重複序列的不穩定延長所引起。這種突變導致有毒RNA的累積和廣泛的細胞功能障礙，影響多個器官系統。

DM1會影響骨骼肌、平滑肌、心臟、眼睛、內分泌系統和中樞神經系統，使其成為臨床表現異質性最高的肌肉萎縮症之一。疾病嚴重程度和發病年齡與CTG重複序列的長度密切相關。

臨床上，DM1 分為三種表現型：

輕型－白內障、輕度肌強直

典型類型－肌肉無力、肌肉僵硬和心臟受累。

先天型－嚴重肌張力低下、呼吸衰竭、發育遲緩。

分子遺傳學檢測可以確診，這對於出現遠端肌肉無力、肌強直和多重器官衰竭的患者來說尤其重要。

目前尚無核准的疾病修正治療，治療重點在於症狀治療和多學科管理，包括心臟功能監測、呼吸支持和輔助治療。

主要亮點

• DM1是一種罕見的慢性進行性疾病。雖然患者人數相對穩定，但呈現逐漸上升趨勢。
• 預計義大利 DM1 患者人數將從 2025 年的 3,800 人逐漸增加到 2035 年的 4,000 人，成長率較低（年複合成長率為 0.4%）。
• 人們對基因療法和RNA標靶療法日益成長的關注有望重塑未來的治療模式。

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

• 我們如何最佳化 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？
• 為確保藥物核准並順利進入市場，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

• Avidity Biosciences, Inc.
• Lupin Ltd.
• PepGen Inc
• Sanofi
• Arrowhead Pharmaceuticals
• Dyne Therapeutics
• Vertex Pharmaceuticals Incorporated
• Harmony Biosciences Management, Inc.

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
• 已確診並接受藥物治療的患者人數
• 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
  • 主要治療方法概述及KOL洞察
• 新興治療方法
  • 重點
  • 值得關注的後期新治療方法－概述、市場上市預期及KOL洞察
  • 值得關注的早期研發管線

第6章：未滿足的需求與TPP分析

• 主要未滿足的需求以及透過新興治療方法實現的未來可能性
• TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

Myotonic Dystrophy Type 1 (DM1) Market Outlook

Thelansis's "Myotonic Dystrophy Type 1 (DM1) 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 Myotonic Dystrophy Type 1 treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Myotonic Dystrophy Type 1 (DM1) Overview

Myotonic Dystrophy Type 1 (DM1) is a multisystemic, autosomal dominant neuromuscular disorder caused by an unstable expansion of a CTG trinucleotide repeat in the 3' untranslated region of the DMPK gene (chromosome 19q13.3). This mutation leads to toxic RNA accumulation and widespread cellular dysfunction affecting multiple organ systems.

DM1 impacts skeletal and smooth muscle, cardiac, ocular, endocrine, and central nervous systems, making it one of the most clinically heterogeneous muscular dystrophies. Disease severity and age of onset correlate strongly with CTG repeat length.

Clinically, DM1 is classified into three phenotypes:

Mild form - cataracts, mild myotonia

Classic form - muscle weakness, myotonia, cardiac involvement

Congenital form - severe hypotonia, respiratory failure, developmental delay

Diagnosis is confirmed through molecular genetic testing, particularly in patients presenting with distal muscle weakness, myotonia, and multisystem involvement

Currently, there are no approved disease-modifying therapies, and management remains symptomatic and multidisciplinary, including cardiac monitoring, respiratory support, and supportive care.

Key Highlights

• DM1 is a rare, chronic, and progressive disorder, with a relatively stable but slowly increasing patient population.
• In Italy, the prevalent DM1 population is expected to grow modestly from 3.8K in 2025 to 4.0K by 2035, reflecting a low growth rate (0.4% CAGR).
• Increasing focus on genetic and RNA-targeted therapies is expected to reshape the future treatment paradigm.

Market Overview

• The DM1 market in Germany is projected to grow from ~$78M in 2025 to ~$245M by 2035, reflecting a strong double-digit CAGR (~12-13%), driven by pipeline innovation and emerging therapies, despite a small patient base.
• The patient population shows minimal growth (~0.4% CAGR), indicating: Stable disease prevalence & Limited impact of incidence changes on market expansion

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/report will cost USD 1,750 per country

Companies Mentioned

• Avidity Biosciences, Inc.
• Lupin Ltd.
• PepGen Inc
• Sanofi
• Arrowhead Pharmaceuticals
• Dyne Therapeutics
• Vertex Pharmaceuticals Incorporated
• Harmony Biosciences Management, Inc.

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)