Leigh Syndrome Market: Latest Study by DelveInsight Forecasts Remarkable Growth During the Forecast Period (2023-32) | PTC Therapeutics (Vatiquinone/EPI-743), Taysha GTx (TSHA-104), Abliva AB (NV354)

Leigh Syndrome Market: Latest Study by DelveInsight Forecasts Remarkable Growth During the Forecast Period (2023-32) | PTC Therapeutics (Vatiquinone/EPI-743), Taysha GTx (TSHA-104), Abliva AB (NV354)
Delveinsight Business Research LLP
The Leigh Syndrome Market is anticipated to evolve immensely in the coming years owing to the growing awareness, increased prevalence, and the anticipated launch of novel therapies in the market.

DelveInsight’s “Leigh Syndrome Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Leigh Syndrome market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).

The report covers emerging Leigh Syndrome drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Leigh Syndrome treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market. 

Leigh Syndrome: An Overview

According to the National Organization for Rare Disorders, Leigh syndrome is a rare genetic neurometabolic disorder. It is characterized by the degeneration of the central nervous system (i.e., brain, spinal cord, and optic nerve).

Signs and symptoms usually progress rapidly. Early symptoms may include poor sucking ability; loss of head control and motor skills; loss of appetite; vomiting; and seizures. As the condition progresses, symptoms may include weakness and lack of muscle tone; spasticity; movement disorders; cerebellar ataxia; and peripheral neuropathy. Complications can lead to impairment of respiratory, heart, and kidney function.

The diagnosis of Leigh syndrome may be confirmed by a thorough clinical evaluation and a variety of specialized tests, particularly advanced imaging techniques. Magnetic resonance imaging (MRI) or computed tomography (CT) scans of the brain may reveal abnormal areas in certain parts of the brain (i.e., basal ganglia, brain stem, and gray matter).

Leigh Syndrome Market Key Facts

  • As per the Genetic and Rare Diseases Information Center (GARD) (2022), the prevalence reported for Leigh syndrome is 1 in 30,000 to 1 in 40,000 people at birth. Mitochondrial DNA-associated Leigh syndrome, which is rarer than nuclear gene-encoded Leigh syndrome, is likely to occur in about 1 in 100,000 to 1 in 140,000 births.

  • As per the People Against Leigh Syndrome (PALS) Registry, one estimate of the incidence of Leigh syndrome is one in 77,000 births or one per 40,000 births for Leigh and Leigh-like disease (a milder version of the syndrome, often not proven by imaging or autopsy).

  • According to the National Organization for Rare Disorders (2022), the classical form of Leigh syndrome develops during infancy (infantile necrotizing encephalopathy) and usually begins between the ages of 3 months and 2 years.

Leigh Syndrome Market

The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Leigh Syndrome pipeline therapies. It also thoroughly assesses the Leigh Syndrome market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.

The report gives complete details of the market trend for each marketed Leigh Syndrome drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.

Leigh Syndrome Epidemiology Assessment 

The epidemiology section provides insights into the historical, current, and forecasted Leigh Syndrome epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Leigh Syndrome epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders. 

The Report Covers the Leigh Syndrome Epidemiology, Segmented as –

  • Total Prevalent Cases of Leigh Syndrome [2019-2032]

  • Onset-specific Prevalence of Leigh Syndrome [2019-2032]

  • Mutation-specific Prevalence of Leigh Syndrome [2019-2032]

Leigh Syndrome Drugs Uptake and Pipeline Development Activities

The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Leigh Syndrome market or expected to be launched during the study period. The analysis covers the Leigh Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Leigh Syndrome drugs based on their sale and market share.

The report also covers the Leigh Syndrome pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Leigh Syndrome companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Learn How the Leigh Syndrome Market Will Evolve and Grow by 2032 @ 


Leigh Syndrome Therapeutics Analysis

Approved disease-specific treatment for LS patients is not currently available, although several new molecules are under investigation. Treatment with several agents like Supplementation with B vitamins, such as thiamine, antiseizure medications, physiotherapy, and pharmacotherapy can slow the progress of the disease and help manage symptoms.

Since the treatment landscape is devoid of any effective curative treatment options, any significant development in this direction is expected to create a tectonic impact on the existing market scenario. 

Several major pharma and biotech companies are developing therapies for Leigh’s disease. Currently, PTC Therapeutics is leading the therapeutics market with its Leigh disease drug candidates in the most advanced stage of clinical development. Some of the drugs in the pipeline include Vatiquinone (PTC Therapeutics), and NV354 (Abliva AB), among others. 

Leigh Syndrome Companies Actively Working in the Therapeutics Market Include

  • PTC Therapeutics

  • Taysha GTx

  • Khondrion

And Many Others

Emerging and Marketed Leigh Syndrome Therapies Covered in the Report Include:

  • Vatiquinone (EPI-743): PTC Therapeutics

  • TSHA-104: Taysha GTx

  • NV354: Abliva AB

And Many Others

Download the Sample Report to Learn More About the Key Companies and Emerging Therapies @


Table of Content (TOC)

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 Patient Population and Epidemiology Trends (In the US, EU5, and Japan)

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 Therapies

12. Leigh Syndrome Emerging Drugs and Latest Therapeutic Advances

13. Leigh Syndrome Seven Major Market Analysis

14. Attribute Analysis

15. Leigh Syndrome Market Outlook (In US, EU5, and Japan)

16. Leigh Syndrome Companies Active in the Market

17. Leigh Syndrome Access and Reimbursement Overview

18. KOL Views on the Leigh Syndrome Market

19. Leigh Syndrome Market Drivers

20. Leigh Syndrome Market Barriers

21. Appendix

22. DelveInsight Capabilities

23. Disclaimer

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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