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市場調查報告書
商品編碼
1449631

全球利氏腦病變治療市場:依疾病類型、藥物類型、年齡層、通路、地區

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

2024年,全球利氏腦病變治療市場規模為2.729億美元,預計2031年將達到4.298億美元,年複合成長率(CAGR)預計將達到6.7%。

報告範圍 報告詳情
基準年 2023年 2023/2024年市場規模 2.729 億美元
實際資料 2019-2023 預測期 2024-2031
預測期2023/2024~2030/2031年複合成長率: 6.70% 2030/2031 年預測值 4.298 億美元
圖 1. Leigh腦病變治療的全球市場佔有率(%)(按地區),2024 年
全球利氏症治療市場-IMG1

Leigh腦病變是一種罕見且嚴重的神經系統疾病,通常始於嬰兒期或幼兒期。此疾病的特徵是精神和運動技能進行進行性喪失,導致嚴重的神經功能障礙,常導致 2 至 3 歲的兒童死亡。然而,有些受影響的個體可能有較溫和的病程,起病較慢,存活期較長。 Leigh症候群具有遺傳異質性,由多種基因突變引起。

  • 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。這意味著每個細胞內該基因的兩個拷貝都帶有突變。

導致利氏症候群的突變通常會損害粒線體(細胞中產生能量的結構)的功能。氧化磷酸化是粒線體能源產出過程的一部分,經常受到損害。結果是廣泛的能量缺乏,特別是影響能量需求高的器官和組織,例如大腦、肌肉和心臟。

市場動態:

由於這種罕見的神經遺傳性疾病(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腦病變治療市場在過去幾年中出現了顯著成長。 Leigh 症候群是一種罕見的粒線體疾病,其特徵是基底節和腦幹雙側病變,主要影響中樞神經系統。它通常在嬰兒期或幼兒期變得明顯,並導致發育遲緩和退化、運動問題、虛弱以及視力和聽力問題。活性化研發力度以開發有效的治療方法以及提高人們對這種嚴重疾病的認知正在推動市場成長。

然而,治療成本上升、缺乏核准的治療方案、缺乏特異性疾病的治療方法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的成長。市場開拓機會包括開發療效提高的新藥、專注於基因治療和粒線體移植研究、與臨床研究人員合作以及新興市場的研發。

本研究的主要特點

  • 本報告對全球利氏腦病變治療市場進行了詳細分析,並列出了以2023年為基準年的預測期(2024-2031)的市場規模和年複合成長率。
  • 它還闡明了不同細分市場的潛在收益成長機會,並解釋了該市場有吸引力的投資提案矩陣。
  • 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、主要企業採取的競爭策略等的主要考察。
  • 它根據以下參數(例如公司亮點、產品系列、主要亮點、績效和策略)介紹了全球利氏腦病變治療市場的主要企業。
  • 本研究涵蓋的主要企業包括 Abliva AB、PTC Therapeutics、VAKS Pharma、MITOCH、Medley Pharmaceuticals Ltd.、Khondrion BV、OMEICOS THERAPEUTICS GMBH、Sumitomo Dainippon Pharma Ltd.、Taysha GTx、Picnic (AllStripes)、 工業。
  • 該報告的見解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。
  • 全球利腦病變治療市場報告迎合了該行業的各個相關人員,如投資者、供應商、產品製造商、經銷商、新進業者和財務分析師。
  • 相關人員可以透過用於分析全球利腦病變治療市場的各種策略矩陣來促進他們的決策。

目錄:

第1章 研究目的與前提

  • 研究目標
  • 先決條件
  • 簡稱

第2章 市場展望

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

第3章市場動態、法規及趨勢分析

  • 市場動態
  • 影響分析
  • 主要亮點
  • 監管場景
  • 產品核准
  • PEST分析
  • 波特的分析
  • 併購場景
  • 主要市場參與者的策略見解

第4章全球利氏腦病變治療市場 - 冠狀病毒(COVID-19)大流行的影響

  • 新型冠狀病毒感染疾病(COVID-19)的流行病學
  • 供需面分析
  • 經濟影響

第5章全球利氏腦病變治療市場,依疾病類型,2019-2031

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

第6章全球利氏腦病變治療市場,依藥物類型,2019-2031

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

第7章全球利氏腦病變治療市場,依給藥途徑,2019-2031

  • 口服
  • 注射
  • 外用
  • 其他

第8章全球利氏腦病變治療市場,依年齡層別,2019-2031

  • 嬰兒
  • 青少年和成人

第9章全球利氏腦病變治療市場,依通路,2019-2031

  • 醫院藥房
  • 零售藥房
  • 網路藥房

第10章全球利氏腦病變治療市場,按地區,2019-2031

  • 北美洲
  • 美國
  • 加拿大
  • 歐洲
  • 英國
  • 德國
  • 義大利
  • 法國
  • 西班牙
  • 俄羅斯
  • 其他歐洲國家
  • 亞太地區
  • 中國
  • 日本
  • 印度
  • ASEAN
  • 澳洲
  • 韓國
  • 其他亞太地區
  • 拉丁美洲
  • 巴西
  • 墨西哥
  • 阿根廷
  • 其他拉丁美洲
  • 中東
  • GCC
  • 以色列
  • 其他中東地區
  • 非洲
  • 北非
  • 中部非洲
  • 南非

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

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