Leigh Syndrome Market to Exhibit Moderate Growth Rate Till 2032, Investigates DelveInsight | Key Companies – Aadi Bioscience, Khondrion, and PTC Therapeutics

Delveinsight Business Research LLP
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 and Share in the 7MM (i.e. the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).

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

Leigh Syndrome: An Overview

Leigh syndrome is a rare, inherited neurodegenerative condition. It usually becomes apparent in infancy, often after a viral infection.

The first signs of Leigh syndrome seen in infancy are usually vomiting, diarrhea, and difficulty swallowing (dysphagia), disrupting eating. These problems often result in an inability to grow and gain weight at the expected rate (failure to thrive). Severe muscle and movement problems are common in Leigh syndrome. 

A thorough clinical evaluation may confirm the diagnosis of Leigh syndrome 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). An MRI uses a magnetic field and radio waves to produce cross-sectional images of particular organs and bodily tissues. During CT scanning, a computer and x-rays are used to create a film showing cross-sectional images of certain tissue structures.

Leigh Syndrome Market Key Facts

As per the Genetic and Rare diseases Information Center (GARD) (n.d.), 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 NORD (n.d.) Leigh syndrome affects males and females in equal numbers.

SURF1 deficiency is a monogenic mitochondrial disorder and is the most common cause of cytochrome c oxidase deficient Leigh syndrome, and approximately 10–15% of people with Leigh syndrome have a SURF1 mutation (Taysha Gene, n.d.).

Leigh Syndrome Market

Leigh Syndrome Market size is expected to increase during the forecast period owing to the rising awareness of this unique manifestation and the expected launch of emerging therapies.

The market outlook section of the report helps to build a detailed comprehension of the historic, current, and forecasted Leigh Syndrome market size by analyzing the impact of current and emerging therapies in the market. It also provides a detailed assessment of the market drivers & barriers, unmet needs, and emerging technologies.

The report gives a thorough detail of the Leigh Syndrome market trend for each marketed drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action (MoA), competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.

Leigh Syndrome Epidemiology

Leigh syndrome (LS) is a mitochondrial disease that typically presents in infancy with subacute neurodegenerative encephalopathy. It is genetically heterogeneous, but mutations in the complex IV assembly genes, particularly SURF1, are an important cause.

The epidemiology section covers insights about the historical and current Leigh Syndrome patient pool and forecasted trends for every seven major countries (7MM)  from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides the diagnosed patient pool and their trends along with assumptions undertaken.  

Leigh Syndrome Epidemiology Segmentation

Total Prevalent/Incident Cases of Leigh Syndrome

Mutation-specific Prevalent/Incident cases of Leigh Syndrome cases (SURF1 and others)

Diagnosed and Treatable cases of SURF1 associated Leigh Syndrome

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Leigh Syndrome Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Leigh Syndrome market or expected to get launched in the market during the study period. The analysis covers Leigh Syndrome market uptake by drugs; patient uptake by therapies; and sales of each drug. 

Report’s Drugs Uptake section helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

The report also covers the Leigh Syndrome Pipeline Development Activities and provides valuable insights about different therapeutic candidates in Phase II, and Phase III stages and the key companies in the market involved in developing targeted therapeutics. It also analyses the recent Development such as collaborations, acquisitions, and mergers, licensing patent details, and other information for emerging therapies.

Leigh Syndrome Therapeutics Analysis

The dynamics of the Leigh Syndrome (SURF1) market is expected to change in the coming years owing to the improvement in research and development (R&D) activities in the therapeutics domain.

Some of the key companies in the Leigh Syndrome Market include:

Aadi Bioscience

Khondrion

PTC Therapeutics

And others

Leigh Syndrome Therapies covered in the report include:

ABI-009

Vatiquinone

Sonlicromanol

And many more.

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Table of Content

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

*The table of contents is not exhaustive; the final content may vary. 

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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 end-to-end comprehensive solutions to improve their performance.

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