Leigh Syndrome Market to Rise by 2034 | PTC Therapeutics, Taysha GTx, Khondrion, and others are expected to boost the market

DelveInsight's "Leigh Syndrome Market Insights, Epidemiology, and Market Forecast-2034" report delivers an in-depth understanding of Leigh Syndrome, historical and forecasted epidemiology as well as the Leigh Syndrome market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

The Leigh Syndrome market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Leigh Syndrome market size from 2020 to 2034, segmented by seven major markets. The Report also covers current Leigh Syndrome treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Leigh Syndrome market.

Key highlights from the leigh syndrome market report:

In December 2023, Abliva received orphan drug designation from the European Commission for NV354, aimed at treating Leigh syndrome.

In June 2014, the US FDA granted orphan drug designation to Vatiquinone for the same condition. With ongoing research and a commitment to advancing treatments, there is hope for developing more effective therapies and ultimately a cure for this complex disorder.

According to DelveInsight, the Leigh syndrome market across the 7MM is projected to undergo significant changes during the study period from 2020 to 2034.

Leigh syndrome is often diagnosed late, resulting in delays in the implementation of necessary interventions. Increasing awareness among healthcare providers and enhancing access to rapid genetic testing could promote earlier diagnosis and treatment.

Throughout the forecast period from 2024 to 2034, the Leigh syndrome market is projected to experience a stable Compound Annual Growth Rate (CAGR). This growth in market revenue is primarily fueled by advancements in diagnostic techniques, heightened awareness of the condition, and an increase in reported cases.

The rising incidence of Leigh syndrome is linked to various genetic mutations, particularly involving key enzymes such as complex I, complex IV, and the pyruvate dehydrogenase complex.

To further propel the Leigh syndrome market in the coming years, companies like Abliva AB and PTC Therapeutics are actively advancing their therapies in the mid to late stages of development. With anticipated approvals of these treatments during the forecast period, the overall therapeutic market for Leigh syndrome is expected to see significant growth.

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Leigh Syndrome Overview

Leigh Syndrome is a rare genetic disorder that primarily affects the central nervous system. It is characterized by progressive neurodegeneration, particularly affecting infants and young children. The syndrome leads to a range of symptoms, including developmental delays and neurological problems.

Causes
Leigh Syndrome is often caused by mutations in genes associated with mitochondrial function, which is crucial for energy production in cells. These mutations can be inherited in an autosomal recessive, autosomal dominant, or mitochondrial pattern. The disorder can be linked to various gene defects, with the most common involving:

MT-ATP6 and MT-ATP8 genes
NDUFS1 and other genes involved in mitochondrial metabolism
Signs and Symptoms
Symptoms of Leigh Syndrome typically appear in the first year of life and may include:

Developmental delays: Slow progression in motor skills and speech
Neurological issues: Seizures, hypotonia (reduced muscle tone), and ataxia (lack of coordination)
Respiratory problems: Difficulty breathing or respiratory failure
Feeding difficulties: Poor appetite or inability to feed properly
Abnormal movements: Chorea (involuntary movements) or dystonia (muscle contractions)
As the condition progresses, symptoms can worsen, leading to severe complications and potentially life-threatening situations.

Diagnosis
Diagnosing Leigh Syndrome involves a combination of clinical evaluation and specialized tests, including:

Clinical history: Reviewing symptoms and family history
MRI scans: Identifying characteristic lesions in the brain
Genetic testing: Confirming mutations in mitochondrial DNA or nuclear genes
Biochemical tests: Assessing mitochondrial function through muscle or blood tests
Treatment Options
Currently, there is no cure for Leigh Syndrome, and treatment focuses on managing symptoms and improving quality of life. Options may include:

Supportive care: Physical, occupational, and speech therapy to assist development
Nutritional support: Special diets or feeding tubes for those with feeding difficulties
Medications: Antiepileptic drugs for seizures and medications to manage other symptoms
Mitochondrial therapies: Investigational treatments, such as coenzyme Q10 or vitamins, aimed at improving mitochondrial function
Research is ongoing to find more effective treatments and better management strategies for individuals with Leigh Syndrome. Early diagnosis and intervention can significantly impact the quality of life and disease progression.

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Leigh Syndrome Market

The Leigh Syndrome market outlook in this report provides a comprehensive understanding of historical, current, and projected market trends. It analyzes the impact of existing therapies on the market, identifies unmet needs, and explores key drivers, barriers, and the demand for improved technology.

This section offers an in-depth analysis of the market trends for each approved drug and late-stage pipeline therapy. It evaluates factors such as annual therapy costs, inclusion and exclusion criteria, mechanisms of action, compliance rates, the growing market need, expanding patient population, targeted patient segments, anticipated launch years, competition with other therapies, brand value, and insights from key opinion leaders. The Leigh Syndrome market data are presented with relevant tables and graphs to provide a clear overview at a glance.

According to DelveInsight, significant changes are expected in the Leigh Syndrome market across the seven major markets (7MM) during the study period from 2020 to 2034.

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Leigh Syndrome Epidemiology

The analysis of Leigh syndrome revealed a prevalence of approximately 1 in 30,000 to 1 in 40,000 individuals at birth. It was observed that 64% of patients with Leigh syndrome had mutations in nuclear DNA, with the SURF1 gene being the most common, identified in 72% of cases among a total of 18 nuclear DNA genes studied.

In Japan, the prevalence of Leigh syndrome was found to be about 1 in 5,000 children. Secondary research indicates that the classical form of Leigh syndrome accounts for nearly 80% of all cases. The epidemiology of Leigh syndrome is anticipated to evolve during the forecast period from 2024 to 2034.

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Leigh Syndrome Drugs Uptake

This section focuses on the uptake rate of the potential Leigh Syndrome drugs recently launched in the Leigh Syndrome market or expected to be launched in 2020-2034. The analysis covers the Leigh Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug.

Leigh Syndrome Drugs Uptake helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on Leigh Syndrome market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Leigh Syndrome Pipeline Development Activities

The Leigh Syndrome report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyzes Leigh Syndrome key players involved in developing targeted therapeutics.

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Leigh Syndrome Therapeutics Assessment

Major key companies are working proactively in the Leigh Syndrome Therapeutics market to develop novel therapies which will drive the Leigh Syndrome treatment markets in the upcoming years.

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Leigh Syndrome Report Key Insights

1. Leigh Syndrome Patient Population
2. Leigh Syndrome Market Size and Trends
3. Key Cross Competition in the Leigh Syndrome Market
4. Leigh Syndrome Market Dynamics (Key Drivers and Barriers)
5. Leigh Syndrome Market Opportunities
6. Leigh Syndrome Therapeutic Approaches
7. Leigh Syndrome Pipeline Analysis
8. Leigh Syndrome Current Treatment Practices/Algorithm
9. Impact of Emerging Therapies on the Leigh Syndrome Market

Table of Contents

1. Key Insights
2. Executive Summary
3. Leigh Syndrome Competitive Intelligence Analysis
4. Leigh Syndrome Market Overview at a Glance
5. Leigh Syndrome Disease Background and Overview
6. Leigh Syndrome Patient Journey
7. Leigh Syndrome Epidemiology and Patient Population
8. Leigh Syndrome Treatment Algorithm, Current Treatment, and Medical Practices
9. Leigh Syndrome Unmet Needs
10. Key Endpoints of Leigh Syndrome Treatment
11. Leigh Syndrome Marketed Products
12. Leigh Syndrome Emerging Therapies
13. Leigh Syndrome Seven Major Market Analysis
14. Attribute Analysis
15. Leigh Syndrome Market Outlook (7 major markets)
16. Leigh Syndrome Access and Reimbursement Overview
17. KOL Views on the Leigh Syndrome Market
18. Leigh Syndrome Market Drivers
19. Leigh Syndrome Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer

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