Summary

The 7 major leigh syndrome markets reached a value of US$ 105.0 Million in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 175.1 Million by 2034, exhibiting a growth rate (CAGR) of 4.76% during 2024-2034.

The Leigh syndrome market has been comprehensively analyzed in IMARC's new report titled " Leigh Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Leigh syndrome is a rare and progressive mitochondrial disorder that typically affects infants and young children. It is caused by mutations in mitochondrial DNA or nuclear DNA that affect the efficiency of mitochondria, which are responsible for generating energy within cells. The symptoms of the ailment can vary widely depending on the specific genetic mutations involved and the age of the onset of disease. However, the most common indications are developmental delays and regression, loss of motor skills, muscle weakness and stiffness, movement coordination challenges, difficulty in swallowing, vision and hearing loss, etc. Several other symptoms include seizures, heart problems, gastrointestinal disorders, metabolic acidosis, etc. The diagnosis is based on a combination of clinical investigations, medical history, and genetic testing. Numerous blood and urine tests are conducted to detect high levels of lactic acid, which can indicate mitochondrial dysfunction. Genetic testing is the gold standard for diagnosis and involves sequencing of the mitochondrial DNA or nuclear genes associated with Leigh syndrome. Additionally, magnetic resonance imaging (MRI) of the brain is utilized to identify characteristic abnormalities in the brainstem and basal ganglia.

The rising cases of rare genetic disorders and the growing unmet clinical need to develop effective therapies and medications to treat such conditions are primarily driving the Leigh syndrome market. In addition to this, the escalating utilization of antioxidant supplements, including coenzyme Q10, vitamin E, vitamin C, etc., to help reduce oxidative stress and inflammation in patients is also bolstering the market growth. Moreover, the widespread adoption of various medications, such as anticonvulsants and muscle relaxants, to manage symptoms of the ailment, including seizures and muscle stiffness, is creating a positive outlook for the market. Apart from this, the emerging popularity of neuroprotective therapies that can prevent or slow down nerve damage and help improve outcomes for patients is further propelling the market growth. Additionally, the rising usage of next-generation sequencing, which can analyze large amounts of DNA quickly and accurately, for identifying mutations in genes that may be missed by other methods is also acting as a significant growth-inducing factor. Moreover, the introduction of targeted therapies that address the underlying mitochondrial dysfunction and novel drugs that target mitochondrial protein synthesis and metabolism is expected to drive the Leigh syndrome market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Leigh syndrome market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for Leigh syndrome and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Leigh syndrome market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the Leigh syndrome market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Leigh syndrome market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Leigh syndrome marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the Leigh syndrome market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the Leigh syndrome market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Leigh syndrome market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the number of prevalent cases (2018-2034) of Leigh syndrome across the seven major markets?
What is the number of prevalent cases (2018-2034) of Leigh syndrome by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Leigh syndrome by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with Leigh syndrome across the seven major markets?
What is the size of the Leigh syndrome patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of Leigh syndrome?
What will be the growth rate of patients across the seven major markets?

Leigh Syndrome: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for Leigh syndrome drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Leigh syndrome market?
What are the key regulatory events related to the Leigh syndrome market?
What is the structure of clinical trial landscape by status related to the Leigh syndrome market?
What is the structure of clinical trial landscape by phase related to the Leigh syndrome market?
What is the structure of clinical trial landscape by route of administration related to the Leigh syndrome market?

ページTOPに戻る

Table of Contents

1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Leigh Syndrome - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence

5 Leigh Syndrome - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Leigh Syndrome - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Diagnosed Cases (2018-2034)
7.2.6 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Diagnosed Cases (2018-2034)
7.3.6 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Diagnosed Cases (2018-2034)
7.4.6 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Diagnosed Cases (2018-2034)
7.5.6 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Diagnosed Cases (2018-2034)
7.6.6 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Diagnosed Cases (2018-2034)
7.7.6 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Diagnosed Cases (2018-2034)
7.8.6 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Diagnosed Cases (2018-2034)
7.9.6 Patient Pool/Treated Cases (2018-2034)

8 Leigh Syndrome - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Leigh Syndrome - Unmet Needs

10 Leigh Syndrome - Key Endpoints of Treatment

11 Leigh Syndrome - Marketed Products
11.1 List of Leigh Syndrome Marketed Drugs Across the Top 7 Markets
11.1.1 Drug Name – Company Name
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets

Kindly note that the complete list of marketed drugs has been provided in the report.

12 Leigh Syndrome - Pipeline Drugs
12.1 List of Leigh Syndrome Pipeline Drugs Across the Top 7 Markets
12.1.1 Vatiquinone - PTC Therapeutics
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 Cysteamine Bitartrate– Horizon Pharma USA, Inc
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Leigh Syndrome - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Leigh Syndrome – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events

15 Leigh Syndrome - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Leigh Syndrome - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Leigh Syndrome - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Leigh Syndrome - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Leigh Syndrome - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Leigh Syndrome - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Leigh Syndrome - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Leigh Syndrome - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Leigh Syndrome - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Leigh Syndrome - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Leigh Syndrome - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Leigh Syndrome - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Leigh Syndrome - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Leigh Syndrome - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Leigh Syndrome - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Leigh Syndrome - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Leigh Syndrome - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Leigh Syndrome - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Leigh Syndrome - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Leigh Syndrome - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Leigh Syndrome - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Leigh Syndrome - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Leigh Syndrome - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Leigh Syndrome - Access and Reimbursement Overview

16 Leigh Syndrome - Recent Events and Inputs From Key Opinion Leaders

17 Leigh Syndrome Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Leigh Syndrome Market – Strategic Recommendations

19 Appendix