肢帶型肌肉萎縮症（LGMD）：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

• 預計到 2035 年，法國 LGMD 市場將從約 1.7 億美元成長到 5.1 億美元。
• 以下因素正在推動市場成長：
• 基因治療發展進展
• 擴大精準診斷和支持性治療的引入範圍。
• 推動市場擴張的主要動力預計將是罕見疾病的高附加價值治療方案。
• 未來的成長取決於基因療法和緩解疾病療法的商業化是否成功。

肢帶型肌肉萎縮症（LGMD）概述

肢帶型肌肉萎縮症（GILOT）是一組高度異質性的遺傳性肌肉疾病，由影響筋膜、肌節或核蛋白的基因突變引起，導致進行性近端肌肉退化。患者表現為肩胛帶和骨盆帶無力、步態蹣跚，以及不同程度的心肺功能受損。層粘蛋白病和肌聚醣病亞型尤其具有較高的心律不整風險，因此需要儘早考慮植入心臟節律器或植入式心律轉復除顫器（ICD）。診斷包括肌酸激酶（CK）水平升高、肌肉切片檢查免疫組化以及基因檢測。雖然目前尚無特效療法，但針對基因型的模式轉移治療正在興起，例如使用腺結合病毒（AAV）的基因療法和糖基化通路調節劑，這些療法目前正在積極評估中。物理治療、矯正器具支撐和心肺監測仍然是基本的治療方法。多學科協作的護理有助於維持患者的功能和生活品質。

主要亮點

• 在法國，預計患有肢帶型肌肉營養不良症的人數將從 2025 年的約 1402 人增加到 2035 年的 1499 人。
• 肢帶型肌肉營養不良症（LGMD）是一種罕見的遺傳性神經肌肉疾病，其特徵是進行性肌肉無力和功能障礙。
• 基因檢測技術的進步使得早期診斷和亞型識別變得更加容易。
• 由於目前尚無廣泛有效的疾病修正治療，因此仍存在巨大的未滿足需求。
• 基因療法和基因醫學的新方法正在改變我們治療疾病的方式。

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

• 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

• Sarepta Therapeutics, Inc.
• ML Bio Solutions, Inc.
• AskBio Inc.
• Atamyo Therapeutics

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

Limb-Girdle Muscular Dystrophy (LGMD) Market Outlook

Thelansis's "Limb-Girdle Muscular Dystrophy (LGMD) 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 Limb-Girdle Muscular Dystrophy treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Limb-Girdle Muscular Dystrophy (LGMD) Overview

Limb-Girdle Muscular Dystrophy is a heterogeneous group of inherited myopathies caused by mutations affecting sarcolemmal, sarcomeric, or nuclear proteins, driving progressive proximal muscle degeneration. Patients present with shoulder and pelvic girdle weakness, waddling gait, and variable cardiac or respiratory involvement; laminopathy and sarcoglycanopathy subtypes carry particular arrhythmia risk requiring early pacemaker or ICD consideration. Diagnosis combines elevated CK, muscle biopsy immunohistochemistry, and genetic panel testing. While no universal cure exists, the paradigm is shifting toward genotype-targeted therapies, including AAV-based gene therapy and glycosylation-pathway modulators under active evaluation. Physiotherapy, orthotic support, and cardiorespiratory surveillance remain foundational. Multidisciplinary care preserves function and quality of life.

Key Highlights

• In France, prevalent LGMD cases are projected to increase from approximately 1,402 in 2025 to 1,499 by 2035.
• LGMD remains a rare genetic neuromuscular disorder associated with progressive muscle weakness and disability.
• Improved genetic testing is facilitating earlier diagnosis and subtype identification.
• Significant unmet need persists due to the absence of broadly effective disease-modifying therapies.
• Emerging gene therapy and genetic medicine approaches are reshaping the treatment landscape.

Market Overview

• The France LGMD market is projected to grow from approximately $170 MN to $510 MN by 2035.
• Market growth is supported by:
• Advancements in gene therapy development
• Increasing adoption of precision diagnostics and supportive care
• Market expansion is expected to be driven by high-value rare disease therapies.
• Future growth will depend on successful commercialization of gene-based and disease-modifying treatments.

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

• Sarepta Therapeutics, Inc.
• ML Bio Solutions, Inc.
• AskBio Inc.
• Atamyo Therapeutics

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)