強直性肌營養不良症第一型(DM1)：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

強直性肌營養不良症第一型（DM1）概述

強直性肌營養不良症第一型（DM1），又稱Steinert氏病，是一種進行性性體染色體顯性遺傳的多系統神經肌肉疾病，由DMPK基因3'非轉譯區不穩定的CTG三核苷酸重複序列擴增所引起。這種擴增導致有毒的轉錄RNA滯留在細胞核內，捕獲肌肉盲樣（MBNL）剪接因子，並在骨骼肌、心肌、平滑肌和中樞神經系統組織中誘發廣泛的胚胎剪接病變。患者表現出典型的臨床症狀，包括肌強直（肌肉鬆弛延遲）、遠端肌無力、眼瞼下垂、白內障、早期脫髮和嚴重的呼吸衰竭。重要的是，由於傳導系統功能障礙可能導致致命的心室心律不整，因此定期進行心電圖檢查和心律調節器監測非常重要。全身麻醉會顯著增加呼吸衰竭和心臟衰竭的風險。診斷需透過標靶基因和分子檢測來確診。截至2026年，治療仍將以症狀治療為主，對於影響日常生活的肌強直，患者需服用美西律，並接受嚴格的循環系統監測；對於通氣不足，則需進行夜間無創通氣治療。然而，隨著包括Zerecimento baciversen（DYNE-101）在內的具有緩解疾病作用的先進RNA標靶治療候選藥物進入III期全球評估以待驗證，治療前景發生顯著變化。

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MicrosoftExcel）
• 流行病學資料（MS Excel，互動式工具）
• 高層洞察（PPT簡報）
• 其他功能：定期更新、自訂和顧問支援。
• 根據 Thelansis 的政策，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）

主要企業

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

目錄

第1章 主要調查結果和分析師說明

• 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
• 依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
• 目前主要治療方法 - 概述及關鍵意見領袖見解
• 新興治療方法
• 重點
• 值得關注的後期新興治療方法 - 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TPP分析

• 主要未滿足需求及新興療法的未來成就
• TPP分析與KOL展望

第7章 監理與償付環境

第8章 附錄

Global Myotonic Dystrophy Type 1 (DM1) Market Outlook

Thelansis's "Global Myotonic Dystrophy Type 1 (DM1) 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 Myotonic Dystrophy Type 1 treatment modalities options for the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America).

Myotonic Dystrophy Type 1 (DM1) Overview

Myotonic dystrophy type 1 (DM1), or Steinert disease, is a progressive, autosomal dominant multisystemic neuromuscular disorder caused by an unstable CTG trinucleotide repeat expansion in the 3' untranslated region of the DMPK gene. This expansion leads to the nuclear retention of toxic transcript RNAs that sequester muscleblind-like (MBNL) splicing factors, inducing widespread embryonic spliceopathy across skeletal, cardiac, smooth muscle, and central nervous tissues. Patients clinically present with classic myotonia (delayed muscle relaxation), distal muscle weakness, ptosis, cataracts, early-onset balding, and severe hypoventilation. Crucially, conduction system disease creates lethal ventricular arrhythmias, making regular electrocardiographic or pacemaker surveillance mandatory. General anesthesia carries an unusually elevated risk of respiratory and cardiac collapse. Diagnosis is confirmed via targeted genetic molecular testing. Management in 2026 remains primarily supportive, utilizing mexiletine for disabling myotonia under strict cardiological monitoring, and nocturnal non-invasive ventilation for hypoventilation. However, the therapeutic landscape has transformed with advanced disease-modifying RNA-targeted candidates, including zeleciment basivarsen (DYNE-101), entering confirmatory pivotal phase 3 global evaluation.

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

• 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)