1 型强直性肌肉营养不良症 (DM1)：市场展望、流行病学、竞争格局、市场预测报告（2025-2035 年）

市场概览

• 儘管德国 DM1 患者数量相对较少，但预计到 2035 年，德国 DM1 市场将从 2025 年的约 7,800 万美元增长到 2035 年的约 2.45 亿美元，呈现两位数的复合年增长率（约 12-13%），这主要得益于研发管线治疗方法和新疗法的出现。
• 患者数量仅略有增加（年复合增长率约为 0.4%），这表明该疾病的流行情况稳定，发病率的变化对市场扩张的影响有限。

1 型强直性肌肉营养不良症 (DM1) 概述

1型强直性肌肉营养不良症（DM1）是一种多系统性常染色体显性遗传神经肌肉疾病，由DMPK基因（19号染色体，q13.3）3'非翻译区中CTG三核苷酸重复序列的不稳定延长所引起。这种突变导致有毒RNA的累积和广泛的细胞功能障碍，影响多个器官系统。

DM1会影响骨骼肌、平滑肌、心臟、眼睛、内分泌系统和中枢神经系统，使其成为临床表现异质性最高的肌肉萎缩症之一。疾病严重程度和发病年龄与CTG重复序列的长度密切相关。

临床上，DM1 分为三种表型：

轻型－白内障、轻度肌强直

典型类型－肌肉无力、肌肉僵硬和心臟受累。

先天型－严重肌张力低下、呼吸衰竭、发育迟缓。

分子遗传学检测可以确诊，这对于出现远端肌肉无力、肌强直和多重器官衰竭的患者来说尤其重要。

目前尚无核准的疾病修正治疗，治疗重点在于症状治疗和多学科管理，包括心臟功能监测、呼吸支持和辅助治疗。

主要亮点

• DM1是一种罕见的慢性进行性疾病。虽然患者人数相对稳定，但呈现逐渐上升趋势。
• 预计义大利 DM1 患者人数将从 2025 年的 3,800 人逐渐增加到 2035 年的 4,000 人，成长率较低（年复合成长率为 0.4%）。
• 人们对基因疗法和RNA标靶疗法日益增长的关注有望重塑未来的治疗模式。

格式化和更新讯息

• 详细报告（PDF）
• 市场预测模型（基于微软Excel）
• 流行病学数据（MS Excel，互动式工具）
• 高阶主管洞察（PPT简报）
• 其他功能：定期更新、自订和顾问支援。
• 根据 Thelansis 的政策，我们确保所有最新更新在发布前都反映在报告内容和市场模型中。

主要问题

• 我们如何优化 G8 市场（美国、欧盟 5 国、日本、中国）的药物开发与生命週期管理策略？
• 从发病率、盛行率、人群组成以及接受药物治疗的患者人数来看，患者数量分别是多少？
• 未来十年市场收入和病患份额的预测是多少？
• 哪些因素对市场趋势影响最大？
• 受访专家对目前和新兴的治疗方法有何看法？
• 哪款在研发线产品最有前景？其上市潜力及未来市场定位如何？
• 主要未被满足的需求是什么？ KOL 对目标受众有何期望？
• 为确保药物核准并顺利进入市场，必须满足哪些关键的监管和支付方要求？

目标国家

• G8
  • 我们
  • EU5
    • 法国
    • 德国
    • 义大利
    • 西班牙
    • 英国
  • 日本
  • 中国

大公司

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

目录

第一章：主要调查结果及分析师说明

• 主要趋势：市场概况、SWOT分析、商业性利益与风险等。

第二章：疾病背景

• 疾病定义、分类、病因和病理生理学、药物标靶等。

第三章：流行病学

• 重点
• 发病率/盛行率
• 已确诊并接受药物治疗的患者人数
• 合併症
• 其他相关患者群

第四章 市场规模及预测

• 重点
• 市场驱动因素与限制因素
• 按药物类别分類的趋势
• 各国具体趋势

第五章 竞争情势

• 目前的治疗方法
  • 重点
  • 诊断和治疗过程/演算法
  • 主要治疗方法概述及KOL洞察
• 新兴治疗方法
  • 重点
  • 值得关注的后期新治疗方法－概述、市场上市预期及KOL洞察
  • 值得关注的早期研发管线

第六章：未满足的需求与TPP分析

• 主要未满足的需求以及透过新兴治疗方法实现的未来可能性
• TPP分析与KOL展望

第七章 监理与报销环境

第八章附录

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)