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市场调查报告书
商品编码
1449631

全球利氏脑病变治疗市场：依疾病类型、药物类型、年龄层、通路、地区

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

2024年，全球利氏脑病变治疗市场规模为2.729亿美元，预计2031年将达到4.298亿美元，年复合成长率（CAGR）预计将达到6.7%。

报告范围 报告详情
基准年 2023年 2023/2024年市场规模 2.729 亿美元
实际资料 2019-2023 预测期 2024-2031
预测期2023/2024~2030/2031年复合成长率： 6.70% 2030/2031 年预测值 4.298 亿美元
图 1. Leigh脑病变治疗的全球市场占有率(%)（按地区），2024 年
全球利氏症治疗市场-IMG1

Leigh脑病变是一种罕见且严重的神经系统疾病，通常始于婴儿期或幼儿期。此疾病的特征是精神和运动技能进行进行性丧失，导致严重的神经功能障碍，常导致 2 至 3 岁的儿童死亡。然而，有些受影响的个体可能有较温和的病程，起病较慢，存活期较长。 Leigh症候群具有遗传异质性，由多种基因突变引起。

  • 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。这意味着每个细胞内该基因的两个拷贝都带有突变。

导致利氏症候群的突变通常会损害粒线体（细胞中产生能量的结构）的功能。氧化磷酸化是粒线体能源产出过程的一部分，经常受到损害。结果是广泛的能量缺乏，特别是影响能量需求高的器官和组织，例如大脑、肌肉和心臟。

市场动态：

由于这种罕见的神经遗传性疾病（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脑病变治疗市场在过去几年中出现了显着增长。 Leigh 症候群是一种罕见的粒线体疾病，其特征是基底节和脑干双侧病变，主要影响中枢神经系统。它通常在婴儿期或幼儿期变得明显，并导致发育迟缓和退化、运动问题、虚弱以及视力和听力问题。活性化研发力度以开发有效的治疗方法以及提高人们对这种严重疾病的认识正在推动市场成长。

然而，治疗成本上升、缺乏核准的治疗方案、缺乏特异性疾病的治疗方法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的成长。市场开拓机会包括开发疗效提高的新药、专注于基因治疗和粒线体移植研究、与临床研究人员合作以及新兴市场的研发。

本研究的主要特点

  • 本报告对全球利氏脑病变治疗市场进行了详细分析，并列出了以2023年为基准年的预测期（2024-2031）的市场规模和复合年增长率。
  • 它还阐明了不同细分市场的潜在收益成长机会，并解释了该市场有吸引力的投资提案矩阵。
  • 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、主要企业采取的竞争策略等的主要考察。
  • 它根据以下参数（例如公司亮点、产品系列、主要亮点、绩效和策略）介绍了全球利氏脑病变治疗市场的主要企业。
  • 本研究涵盖的主要企业包括 Abliva AB、PTC Therapeutics、VAKS Pharma、MITOCH、Medley Pharmaceuticals Ltd.、Khondrion BV、OMEICOS THERAPEUTICS GMBH、Sumitomo Dainippon Pharma Ltd.、Taysha GTx、Picnic (AllStripes)、 工业。
  • 该报告的见解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。
  • 全球利脑病变治疗市场报告迎合了该行业的各个相关人员，如投资者、供应商、产品製造商、经销商、新进业者和财务分析师。
  • 相关人员可以透过用于分析全球利脑病变治疗市场的各种策略矩阵来促进他们的决策。

目录：

第一章 研究目的与前提

  • 研究目标
  • 先决条件
  • 简称

第二章 市场展望

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

第三章市场动态、法规及趋势分析

  • 市场动态
  • 影响分析
  • 主要亮点
  • 监管场景
  • 产品核准
  • PEST分析
  • 波特的分析
  • 併购场景
  • 主要市场参与者的策略见解

第四章全球利氏脑病变治疗市场 - 冠状病毒（COVID-19）大流行的影响

  • 新型冠状病毒感染疾病（COVID-19）的流行病学
  • 供需面分析
  • 经济影响

第五章全球利氏脑病变治疗市场，依疾病类型，2019-2031

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

第六章全球利氏脑病变治疗市场，依药物类型，2019-2031

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

第七章全球利氏脑病变治疗市场，依给药途径，2019-2031

  • 口服
  • 注射
  • 外用
  • 其他的

第8章全球利氏脑病变治疗市场，依年龄组别，2019-2031

  • 婴儿
  • 青少年和成人

第9章全球利氏脑病变治疗市场，依通路，2019-2031

  • 医院药房
  • 零售药房
  • 网路药房

第10章全球利氏脑病变治疗市场，按地区，2019-2031

  • 北美洲
  • 美国
  • 加拿大
  • 欧洲
  • 英国
  • 德国
  • 义大利
  • 法国
  • 西班牙
  • 俄罗斯
  • 其他欧洲国家
  • 亚太地区
  • 中国
  • 日本
  • 印度
  • ASEAN
  • 澳洲
  • 韩国
  • 其他亚太地区
  • 拉丁美洲
  • 巴西
  • 墨西哥
  • 阿根廷
  • 其他拉丁美洲
  • 中东
  • GCC
  • 以色列
  • 其他中东地区
  • 非洲
  • 北非
  • 中部非洲
  • 南非

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12章 章节

  • 调查方法
  • 关于出版商
简介目录
Product Code: CMI6644

The global Leigh syndrome treatment market size was valued at US$ 272.9 Million in 2024 and is expected to reach US$ 429.8 Million by 2031, growing at a Compound Annual Growth Rate (CAGR) of 6.7% from 2024 to 2031.

Report Coverage Report Details
Base Year: 2023 Market Size in 2023/2024: US$ 272.9 Mn
Historical Data for: 2019 to 2023 Forecast Period: 2024 - 2031
Forecast Period 2023/2024 to 2030/2031 CAGR: 6.70% 2030/2031 Value Projection: US$ 429.8 Mn
Figure 1. Global Leigh Syndrome Treatment Market Share (%), By Region, 2024
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 (2024-2031), considering 2023 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, 2019-2031, (US$ Million)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020-2031
    • Segment Trends
  • Mitochondrial Complex I Deficiency
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Mitochondrial Complex III Deficiency
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Mitochondrial Complex IV Deficiency
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • LSAD
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Other
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)

6. Global Leigh Syndrome Treatment Market, By Drug Type, 2019-2031, (US$ Million)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020-2031
    • Segment Trends
  • Vitamin Supplements
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Antioxidants
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Cannabidiol
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Anticonvulsants
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Others
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)

7. Global Leigh Syndrome Treatment Market, By Route of Administration, 2019-2031, (US$ Million)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020-2031
    • Segment Trends
  • Oral
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Injectable
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Topical
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Others
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)

8. Global Leigh Syndrome Treatment Market, By Age Group, 2019-2031, (US$ Million)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020-2031
    • Segment Trends
  • Infants
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Teenagers and Adults
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)

9. Global Leigh Syndrome Treatment Market, By Distribution Channel, 2019-2031, (US$ Million)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020-2031
    • Segment Trends
  • Hospital Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Retail Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)
  • Online Pharmacies
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, 2019-2031, (US$ Million)

10. Global Leigh Syndrome Treatment Market, By Region, 2019-2031, (US$ Million)

  • Introduction
    • Market Share Analysis, 2024 and 2031 (%)
    • Y-o-Y Growth Analysis, 2020-2031
    • Segment Trends
  • North America
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031, (US$ Million)
  • U.S.
  • Canada
  • Europe
    • Introduction
    • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031, (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, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Route of Administration, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Age Group, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031, (US$ Million)
    • Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031, (US$ Million)
  • China
  • Japan
  • India
  • ASEAN
  • Australia
  • South Korea
  • Rest of Asia Pacific
  • Latin America
  • Brazil
  • Mexico
  • Argentina
  • Rest of Latin America
  • Middle East
  • GCC
  • Israel
  • Rest of Middle East
  • Africa
  • North Africa
  • Central Africa
  • South Africa

11. Competitive Landscape

  • 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

12. Section

  • Research Methodology
  • About us