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市場調查報告書
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1705809

全球 Leigh 綜合症治療市場按疾病類型、藥物類型、年齡層、分銷管道和地區分類

Global Leigh Syndrome Treatment Market, By Disease Type, By Drug Type, By Age Group, By Distribution Channel, By Geography
出版日期: | 出版商: Coherent Market Insights | 英文 172 Pages | 商品交期: 2-3個工作天內

價格
簡介目錄

2025 年全球 Leigh 症候群治療市場規模估計為 2.733 億美元，預計到 2032 年將達到 4.36 億美元，2025 年至 2032 年的複合年成長率為 6.9%。

報告範圍 報告詳細資訊
基準年 2024 2025年的市場規模 2.733億美元
效能數據 從2020年到2024年 預測期 2025年至2032年
預測期：2025-2032年複合年成長率： 6.90% 2032年價值預測 4.36億美元
數字。 2025 年 Leigh 症候群治療的全球市場佔有率（按地區分類）
全球Leigh症候群治療市場-IMG1

Leigh 症候群，也稱為 Leigh腦病變，是一種罕見且嚴重的神經系統疾病，通常發病於嬰兒期或幼兒期。此疾病的特徵是精神和運動技能進行性喪失，導致嚴重的神經系統殘疾，並經常在 2-3 歲時死亡。然而，一些受影響的個體可能會經歷較輕的病程、較晚的發病時間和較長的生存期。 Leigh 症候群在遺傳上具有異質性，是由多種基因突變引起的。

  • 粒線體 DNA 突變：粒線體 DNA (mtDNA) 是母系遺傳的（僅從母親遺傳）。
  • 核 DNA 突變：更常見的是，以體染色體隱性方式遺傳的核 DNA 基因突變會導致 Leigh 症候群。這意味著每個細胞中該基因的兩個副本都攜帶突變。

導致 Leigh 症候群的基因突變通常會損害細胞中產生能量的結構—粒線體的功能。在許多情況下，氧化磷酸化（粒線體能源產出過程的一部分）會發生故障。這種疾病會導致廣泛的能量缺乏，特別是影響大腦、肌肉和心臟等能量需求高的器官和組織。

市場動態：

由於這種罕見的神經遺傳疾病（也稱為亞急性壞死性脊髓型頸椎病變或 Leigh 病）的盛行率不斷上升，全球 Leigh 症候群治療市場在過去幾年中經歷了顯著成長。 Leigh 症候群是一種罕見疾病，其特徵是基底核和腦幹的雙側病變，主要影響中樞神經系統，是一種粒線體疾病。它通常在嬰兒期或幼兒期變得明顯，並導致發育遲緩或倒退、運動問題、虛弱以及視力和聽力問題。為開發有效治療方法而進行的研究活動活性化，以及人們對這種嚴重疾病的認知不斷提高，正在推動市場成長。

然而，治療費用高、核准的治療方案少、缺乏特異性疾病的治療以及診斷這種罕見遺傳疾病的困難等因素可能會限制市場成長。市場相關人員面臨各種商業機會，包括開發療效更高的新藥、專注於基因療法和粒線體移植研究、與臨床研究人員合作以及在新興市場發展。

本研究的主要特點

  • 本研究報告對全球 Leigh 症候群治療市場進行了詳細分析，並以 2024 年為基準年，展示了預測期（2025-2032 年）的市場規模和復合年成長率。
  • 它還強調了不同領域的潛在收益成長機會，並說明了該市場的引人注目的投資提案矩陣。
  • 它還提供了有關市場促進因素、限制因素、機會、新產品發布和核准、市場趨勢、區域前景和主要企業採用的競爭策略的主要考察。
  • 全球 Leigh 症候群治療市場的主要企業根據以下參數進行分析，例如公司亮點、產品系列、關鍵亮點、績效和策略。
  • 研究中介紹的主要企業包括 Abliva AB、PTC Therapeutics、VAKS Pharma、MITOCH、Medley Pharmaceuticals Ltd.、Khondrion BV 和 OMEICOS THERAPEUTICS GMBH。住友大日本製藥住友大日本製藥有限公司、Taysha GTx、PicnicHealth (AllStripes)、工業, Inc.、Sarepta Therapeutics, Inc. 和 MECOSON LABS PRIVATE LIMITED。
  • 本報告的見解將使負責人和公司經營團隊能夠就未來的產品發布、類型升級、市場擴張和行銷策略做出明智的決策。
  • 全球 Leigh 症候群治療市場報告迎合了該行業的各個相關人員，例如投資者、供應商、產品製造商、經銷商、新進業者和金融分析師。
  • 相關人員可以透過用於分析全球 Leigh 症候群治療市場的各種策略矩陣來簡化決策。

目錄：

第1章 調查目的與前提條件

  • 研究目標
  • 先決條件
  • 簡稱

第2章 市場展望

  • 報告描述
    • 市場定義和範圍
  • 執行摘要
  • Coherent Opportunity Map（COM）

第3章市場動態、法規與趨勢分析

  • 市場動態
  • 影響分析
  • 主要亮點
  • 監管情景
  • 產品核可
  • PEST分析
  • 波特分析
  • 併購場景
  • 來自主要市場參與者的策略見解

4.全球Leigh 症候群治療市場-冠狀病毒 (COVID-19) 大流行的影響

  • COVID-19流行病學
  • 供需側分析
  • 經濟影響

第 5 章 Leigh 症候群治療市場（按疾病分類），2020 年至 2032 年

  • 粒線體複合物I缺乏症
  • 粒線體複合物 III 缺乏症
  • 粒線體複合物IV缺乏症
  • LSAD
  • 其他

6. 2020 年至 2032 年 Leigh 症候群治療市場（按藥物）

  • 維生素補充劑
  • 抗氧化劑
  • 大麻二酚
  • 抗驚厥藥
  • 其他

7. 2020 年至 2032 年 Leigh 症候群治療全球市場（依給藥途徑）

  • 口服
  • 注射
  • 話題
  • 其他

8. 2020 年至 2032 年全球 Leigh 症候群治療市場（依年齡族群）

  • 嬰兒
  • 青少年和成年人

9. 全球Leigh症候群治療市場（依通路分類），2020-2032年

  • 醫院藥房
  • 零售藥局
  • 網路藥局

第 10 章 Leigh 症候群治療市場（按地區分類），2020 年至 2032 年

  • 北美洲
  • 歐洲
  • 亞太地區
  • 拉丁美洲
  • 中東
  • 非洲

第11章競爭格局

  • Abliva AB
  • PTC Therapeutics
  • VAKS Pharma
  • MITOCH
  • Medley Pharmaceuticals Ltd.
  • Khondrion BV
  • OMEICOS THERAPEUTICS GMBH.
  • Edison Pharmaceuticals, Inc.
  • Dainippon Sumitomo Pharma Co. Ltd.
  • Taysha GTx
  • PicnicHealth（AllStripes）
  • Takeda Pharmaceutical Company
  • Biogen
  • Ionis Pharmaceuticals, Inc.
  • Sarepta Therapeutics, Inc.
  • MECOSON LABS PRIVATE LIMITED

第12章 章節

  • 調查方法
  • 關於出版商
簡介目錄
Product Code: CMI6644

Global Leigh Syndrome Treatment Market is estimated to be valued at USD 273.3 Mn in 2025 and is expected to reach USD 436.0 Mn by 2032, growing at a compound annual growth rate (CAGR) of 6.9% from 2025 to 2032.

Report Coverage Report Details
Base Year: 2024 Market Size in 2025: USD 273.3 Mn
Historical Data for: 2020 To 2024 Forecast Period: 2025 To 2032
Forecast Period 2025 to 2032 CAGR: 6.90% 2032 Value Projection: USD 436.0 Mn
Figure. Global Leigh Syndrome Treatment Market Share (%), By Region 2025
Global Leigh Syndrome Treatment Market - IMG1

Leigh syndrome, also known as Leigh disease, is a rare, severe neurological disorder that typically becomes apparent in infancy or early childhood. It is characterized by progressive loss of mental and movement abilities, leading to severe neurological disability and often death by two to three years of age. However, some affected individuals may have a milder course of the disease with later onset and longer survival. Leigh syndrome is genetically heterogeneous, meaning it can be caused by mutations in various genes, and it can be inherited in several different ways, depending on the gene involved:

  • Mitochondrial DNA Mutations: Some cases of Leigh syndrome are caused by mutations in genes found within mitochondrial DNA (mtDNA), which is inherited maternally (only passed down from mothers)
  • Nuclear DNA Mutations: More commonly, the mutations associated with Leigh syndrome are in genes located on nuclear DNA, which is inherited in an autosomal recessive pattern. This means that both copies of the gene in each cell have mutations.

The mutations responsible for Leigh syndrome typically impair the function of mitochondria, the energy-producing structures within cells. This often involves disruptions in oxidative phosphorylation, which is part of the mitochondrial energy generation process. The disruption results in widespread energy deficiencies, particularly affecting organs and tissues with high energy demands such as the brain, muscle, and heart.

Market Dynamics:

The global Leigh syndrome treatment market has been witnessing notable growth over the past few years owing to the rising prevalence of this rare neurological genetic disorder that is also referred to as subacute necrotizing encephalomyelopathy or Leigh disease. Leigh syndrome is a rare disorder characterized by bilateral lesions in the basal ganglia and brainstem that primarily affects the central nervous system and is a mitochondrial disease. It usually becomes evident in infancy or early childhood and can cause developmental delays or regression, movement problems, weakness, vision/hearing problems, and others. Increasing research activities to develop effective treatment options and rising awareness about this serious illness are fueling the market growth.

However, the market growth can be limited due to factors like high cost of treatment, availability of few approved treatment options, lack of disease-specific treatment, and difficulty in diagnosing this rare genetic disorder. Various opportunities for market players lie in developing novel drugs with improved efficacy, focusing on gene therapy and mitochondrial transplantation research, collaborating with clinical researchers, and expanding in emerging markets.

Key Features of the Study:

  • This report provides an in-depth analysis of the global Leigh syndrome treatment market, and provides market size (US$ Million) and Compound Annual Growth Rate (CAGR %) for the forecast period (2025-2032), considering 2024 as the base year
  • It elucidates potential revenue growth opportunities across different segments and explains attractive investment proposition matrices for this market
  • This study also provides key insights about market drivers, restraints, opportunities, new product launches or approvals, market trends, regional outlook, and competitive strategies adopted by key players
  • It profiles key players in the global Leigh syndrome treatment market based on the following parameters such as company highlights, products portfolio, key highlights, financial performance, and strategies
  • The key companies covered as a part of this study include Abliva AB, PTC Therapeutics, VAKS Pharma, MITOCH, Medley Pharmaceuticals Ltd., Khondrion BV, OMEICOS THERAPEUTICS GMBH. , Edison Pharmaceuticals, Inc. , Dainippon Sumitomo Pharma Co. Ltd., Taysha GTx, PicnicHealth (AllStripes), Takeda Pharmaceutical Company , Biogen, Ionis Pharmaceuticals, Inc., Sarepta Therapeutics, Inc., and MECOSON LABS PRIVATE LIMITED
  • Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
  • The global Leigh syndrome treatment market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
  • Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global Leigh syndrome treatment market

Detailed Segmentation:

  • By Disease Type:
    • Mitochondrial Complex I Deficiency
    • Mitochondrial Complex III Deficiency
    • Mitochondrial Complex IV Deficiency
    • LSAD
    • Others
  • By Disease Type:
    • Vitamin Supplements
    • Antioxidants
    • Cannabidiol
    • Anticonvulsants
    • Others (Late Phase Drugs, etc.)
  • By Route of Administration:
    • Oral
    • Injectable
    • Topical
    • Others
  • By Age Group:
    • Infants
    • Teenagers and Adults
  • By Distribution Channel:
    • Hospital Pharmacies
    • Retail pharmacies
    • Online Pharmacies
  • By Region:
    • North America
    • Europe
    • Asia Pacific
    • Latin America
    • Middle East
    • Africa
  • Company Profiles:
    • Abliva AB
    • PTC Therapeutics
    • VAKS Pharma
    • MITOCH
    • Medley Pharmaceuticals Ltd.
    • Khondrion BV
    • OMEICOS THERAPEUTICS GMBH.
    • Edison Pharmaceuticals, Inc.
    • Dainippon Sumitomo Pharma Co. Ltd.
    • Taysha GTx
    • PicnicHealth (AllStripes)
    • Takeda Pharmaceutical Company
    • Biogen
    • Ionis Pharmaceuticals, Inc.
    • Sarepta Therapeutics, Inc.
    • MECOSON LABS PRIVATE LIMITED

Table of Contents:

1. Research Objectives and Assumptions

  • Research Objectives
  • Assumptions
  • Abbreviations

2. Market Purview

  • Report Description
    • Market Definition and Scope
  • Executive Summary
    • Global Leigh Syndrome Treatment Market, By Disease Type
    • Global Leigh Syndrome Treatment Market, By Drug Type
    • Global Leigh Syndrome Treatment Market, By Route of Administration
    • Global Leigh Syndrome Treatment Market, By Age Group
    • Global Leigh Syndrome Treatment Market, By Distribution Channel
    • Global Leigh Syndrome Treatment Market, By Region
  • Coherent Opportunity Map (COM)

3. Market Dynamics, Regulations, and Trends Analysis

  • Market Dynamics
    • Drivers
    • Restraints
    • Opportunities
  • Impact Analysis
  • Key Highlights
  • Regulatory Scenario
  • Product Approvals
  • PEST Analysis
  • PORTER's Analysis
  • Merger and Acquisition Scenario
  • Strategic Insights by Key Market Players

4. Global Leigh Syndrome Treatment Market - Impact of Coronavirus (COVID-19) Pandemic

  • COVID-19 Epidemiology
  • Supply Side and Demand Side Analysis
  • Economic Impact

5. Global Leigh Syndrome Treatment Market, By Disease Type, 2020-2032, (US$ Million)

  • Introduction
    • Market Share Analysis, 2025 and 2032 (%)
    • Y-o-Y Growth Analysis, 2021-2032
    • Segment Trends
  • Mitochondrial Complex I Deficiency
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Mitochondrial Complex III Deficiency
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Mitochondrial Complex IV Deficiency
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • LSAD
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Other
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)

6. Global Leigh Syndrome Treatment Market, By Drug Type, 2020-2032, (US$ Million)

  • Introduction
    • Market Share Analysis, 2025 and 2032 (%)
    • Y-o-Y Growth Analysis, 2021-2032
    • Segment Trends
  • Vitamin Supplements
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Antioxidants
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Cannabidiol
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Anticonvulsants
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Others
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)

7. Global Leigh Syndrome Treatment Market, By Route of Administration, 2020-2032, (US$ Million)

  • Introduction
    • Market Share Analysis, 2025 and 2032 (%)
    • Y-o-Y Growth Analysis, 2021-2032
    • Segment Trends
  • Oral
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Injectable
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Topical
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Others
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)

8. Global Leigh Syndrome Treatment Market, By Age Group, 2020-2032, (US$ Million)

  • Introduction
    • Market Share Analysis, 2025 and 2032 (%)
    • Y-o-Y Growth Analysis, 2021-2032
    • Segment Trends
  • Infants
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Teenagers and Adults
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)

9. Global Leigh Syndrome Treatment Market, By Distribution Channel, 2020-2032, (US$ Million)

  • Introduction
    • Market Share Analysis, 2025 and 2032 (%)
    • Y-o-Y Growth Analysis, 2021-2032
    • Segment Trends
  • Hospital Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Retail Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)
  • Online Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2020-2032, (US$ Million)

10. Global Leigh Syndrome Treatment Market, By Region, 2020-2032, (US$ Million)

  • Introduction
    • Market Share Analysis, 2025 and 2032 (%)
    • Y-o-Y Growth Analysis, 2021-2032
    • Segment Trends
  • North America
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032, (US$ Million)
    • U.S.
    • Canada
  • Europe
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032, (US$ Million)
    • U.K.
    • Germany
    • Italy
    • France
    • Spain
    • Russia
    • Rest of Europe
  • Asia Pacific
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032, (US$ Million)
    • China
    • Japan
    • India
    • ASEAN
    • Australia
    • South Korea
    • Rest of Asia Pacific
  • Latin America
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032, (US$ Million)
    • Brazil
    • Mexico
    • Argentina
    • Rest of Latin America
  • Middle East
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2020-2032, (US$ Million)
    • GCC
    • Israel
    • Rest of Middle East
  • Africa
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2020-2032, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country/Region, 2020-2032, (US$ Million)
    • North Africa
    • Central Africa
    • South Africa

11. Competitive Landscape

  • Abliva AB
    • Company Highlights
    • Product Portfolio
    • Key Developments
    • Financial Performance
    • Strategies
  • PTC Therapeutics
    • Company Highlights
    • Product Portfolio
    • Key Developments
    • Financial Performance
    • Strategies
  • VAKS Pharma
    • Company Highlights
    • Product Portfolio
    • Key Developments
    • Financial Performance
    • Strategies
  • MITOCH
    • Company Highlights
    • Product Portfolio
    • Key Developments
    • Financial Performance
    • Strategies
  • Medley Pharmaceuticals Ltd.
    • Company Highlights
    • Product Portfolio
    • Key Developments
    • Financial Performance
    • Strategies
  • Khondrion BV
    • Company Highlights
    • Product Portfolio
    • Key Developments
    • Financial Performance
    • Strategies
  • OMEICOS THERAPEUTICS GMBH.
    • Company Highlights
    • Product Portfolio
    • Key Developments
    • Financial Performance
    • Strategies
  • Edison Pharmaceuticals, Inc.
  • Dainippon Sumitomo Pharma Co. Ltd.
  • Taysha GTx
  • PicnicHealth (AllStripes)
  • Takeda Pharmaceutical Company
  • Biogen
  • Ionis Pharmaceuticals, Inc.
  • Sarepta Therapeutics, Inc.
  • MECOSON LABS PRIVATE LIMITED

12. Section

  • Research Methodology
  • About us