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市场调查报告书
商品编码
1705809

全球 Leigh 综合症治疗市场按疾病类型、药物类型、年龄层、分销管道和地区划分

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个工作天内

价格
简介目录

2025 年全球 Leigh 症候群治疗市场规模估计为 2.733 亿美元，预计到 2032 年将达到 4.36 亿美元，2025 年至 2032 年的复合年增长率为 6.9%。

报告范围 报告详细信息
基准年 2024 2025年的市场规模 2.733亿美元
效能数据 从2020年到2024年 预测期 2025年至2032年
预测期：2025-2032年复合年增长率： 6.90% 2032年价值预测 4.36亿美元
数字。 2025 年 Leigh 症候群治疗的全球市场占有率（按地区划分）
全球Leigh症候群治疗市场-IMG1

Leigh 综合征，也称为 Leigh脑病变，是一种罕见且严重的神经系统疾病，通常发病于婴儿期或幼儿期。此疾病的特征是精神和运动技能进行性丧失，导致严重的神经系统残疾，并经常在 2-3 岁时死亡。然而，一些受影响的个体可能会经历较轻的病程、较晚的发病时间和较长的生存期。 Leigh 症候群在遗传上具有异质性，是由多种基因突变引起的。

  • 粒线体 DNA 突变：粒线体 DNA (mtDNA) 是母系遗传的（仅从母亲遗传）。
  • 核 DNA 突变：更常见的是，以体染色体隐性方式遗传的核 DNA 基因突变会导致 Leigh 症候群。这意味着每个细胞中该基因的两个副本都携带突变。

导致 Leigh 症候群的基因突变通常会损害细胞中产生能量的结构—粒线体的功能。在许多情况下，氧化磷酸化（粒线体能源产出过程的一部分）会发生故障。这种疾病会导致广泛的能量缺乏，特别是影响大脑、肌肉和心臟等能量需求高的器官和组织。

市场动态：

由于这种罕见的神经遗传疾病（也称为亚急性坏死性脊髓型颈椎病变或 Leigh 病）的盛行率不断上升，全球 Leigh 症候群治疗市场在过去几年中经历了显着增长。 Leigh 症候群是一种罕见疾病，其特征是基底核和脑干的双侧病变，主要影响中枢神经系统，是一种粒线体疾病。它通常在婴儿期或幼儿期变得明显，并导致发育迟缓或倒退、运动问题、虚弱以及视力和听力问题。为开发有效治疗方法而进行的研究活动活性化，以及人们对这种严重疾病的认识不断提高，正在推动市场成长。

然而，治疗费用高、核准的治疗方案少、缺乏特异性疾病的治疗以及诊断这种罕见遗传疾病的困难等因素可能会限制市场成长。市场相关人员面临各种商业机会，包括开发疗效更高的新药、专注于基因疗法和粒线体移植研究、与临床研究人员合作以及在新兴市场发展。

本研究的主要特点

  • 本研究报告对全球 Leigh 症候群治疗市场进行了详细分析，并以 2024 年为基准年，展示了预测期（2025-2032 年）的市场规模和復合年增长率。
  • 它还强调了不同领域的潜在收益成长机会，并说明了该市场的引人注目的投资提案矩阵。
  • 它还提供了有关市场驱动因素、限制因素、机会、新产品发布和核准、市场趋势、区域前景和主要企业采用的竞争策略的主要考察。
  • 全球 Leigh 综合征治疗市场的主要企业根据以下参数进行分析，例如公司亮点、产品系列、关键亮点、绩效和策略。
  • 研究中介绍的主要企业包括 Abliva AB、PTC Therapeutics、VAKS Pharma、MITOCH、Medley Pharmaceuticals Ltd.、Khondrion BV 和 OMEICOS THERAPEUTICS GMBH。住友大日本製药住友大日本製药有限公司、Taysha GTx、PicnicHealth (AllStripes)、工业, Inc.、Sarepta Therapeutics, Inc. 和 MECOSON LABS PRIVATE LIMITED。
  • 本报告的见解将使负责人和公司经营团队能够就未来的产品发布、类型升级、市场扩张和行销策略做出明智的决策。
  • 全球 Leigh 综合征治疗市场报告迎合了该行业的各个相关人员，例如投资者、供应商、产品製造商、经销商、新进业者和金融分析师。
  • 相关人员可以透过用于分析全球 Leigh 症候群治疗市场的各种策略矩阵来简化决策。

目录：

第一章 调查目的与前提条件

  • 研究目标
  • 先决条件
  • 简称

第二章 市场展望

  • 报告描述
    • 市场定义和范围
  • 执行摘要
  • Coherent Opportunity Map（COM）

第三章市场动态、法规与趋势分析

  • 市场动态
  • 影响分析
  • 主要亮点
  • 监管情景
  • 产品核可
  • PEST分析
  • 波特分析
  • 併购场景
  • 来自主要市场参与者的策略见解

4. 全球 Leigh 症候群治疗市场-冠状病毒 (COVID-19) 大流行的影响

  • COVID-19流行病学
  • 供需侧分析
  • 经济影响

第 5 章 Leigh 症候群治疗市场（按疾病划分），2020 年至 2032 年

  • 粒线体复合物I缺乏症
  • 粒线体复合物 III 缺乏症
  • 粒线体复合物IV缺乏症
  • LSAD
  • 其他的

6. 2020 年至 2032 年 Leigh 症候群治疗市场（按药物）

  • 维生素补充剂
  • 抗氧化剂
  • 大麻二酚
  • 抗惊厥药
  • 其他的

7. 2020 年至 2032 年 Leigh 症候群治疗全球市场（依给药途径）

  • 口服
  • 注射
  • 话题
  • 其他的

8. 2020 年至 2032 年全球 Leigh 症候群治疗市场（依年龄族群）

  • 婴儿
  • 青少年和成年人

9. 全球Leigh症候群治疗市场（依通路划分），2020-2032年

  • 医院药房
  • 零售药局
  • 网路药局

第 10 章 Leigh 症候群治疗市场（按地区划分），2020 年至 2032 年

  • 北美洲
  • 欧洲
  • 亚太地区
  • 拉丁美洲
  • 中东
  • 非洲

第十一章竞争格局

  • 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

第十二章 章节

  • 调查方法
  • 关于出版商
简介目录
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