Leigh Syndrome Market Report 2034: Epidemiology, Pipeline Therapies, Latest FDA, EMA, PDMA Approvals by DelveInsight

Leigh Syndrome Market Insight Report

Leigh Syndrome companies are NV354, and others.

LAS VEGAS, NEVADA, UNITED STATES, July 11, 2024 /EINPresswire.com/ -- DelveInsight’s “Leigh Syndrome Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Leigh Syndrome, historical and forecasted epidemiology as well as the Leigh Syndrome market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

To Know in detail about the Leigh Syndrome market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Leigh Syndrome Market Forecast

Some of the key facts of the Leigh Syndrome Market Report:
The Leigh Syndrome market size is anticipated to grow with a significant CAGR during the study period (2020-2034).
Leigh syndrome is often diagnosed late, leading to delays in initiating appropriate interventions. Improved awareness among healthcare providers and access to rapid genetic testing could facilitate earlier diagnosis and intervention.
Throughout the forecast period (2024-2034), the Leigh syndrome market is expected to maintain a stable Compound Annual Growth Rate (CAGR). This increase in market revenue is primarily driven by progress in diagnostic methodologies, increased awareness of the condition, and a rising prevalence of reported cases.
The cases of Leigh syndrome are increasing due to various genetic mutations. Common enzymes associated with Leigh syndrome include complex I, complex IV, and pyruvate dehydrogenase complex.
To drive the Leigh syndrome market in future years, several companies such as Abliva AB, PTC Therapeutics, and others are developing their assets in the mid-late stage of development. With the expected approval of these therapies during the forecast period [2024–2034], the overall therapeutic market of Leigh syndrome is likely to witness a rise at a significant CAGR
Key Leigh Syndrome Companies: NV354, and others
Key Leigh Syndrome Therapies: Abliva AB, and others
In December 2023, Abliva was granted orphan drug designation by the European Commission for NV354, intended for the treatment of Leigh syndrome.
In June 2014, the US FDA granted orphan drug designation to Vatiquinone for the treatment of Leigh Syndrome.
With ongoing research and continued dedication, the future holds hope for even more effective treatments and, ultimately, a cure for this challenging condition. According to DelveInsight, the Leigh syndrome market in the 7MM is expected to change significantly during the study period 2020–2034.
The Leigh Syndrome market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Leigh Syndrome pipeline products will significantly revolutionize the Leigh Syndrome market dynamics.

Leigh Syndrome Overview
Leigh syndrome is a rare genetic neurometabolic disorder characterized by the progressive degeneration of the central nervous system, including the brain, spinal cord, and optic nerve. The inheritance pattern of Leigh syndrome is typically autosomal recessive, although X-linked recessive and maternal inheritance due to mitochondrial DNA mutations are also observed.

Key enzymes involved in Leigh syndrome include complex I, complex IV, and the pyruvate dehydrogenase complex. Dysfunction in coenzyme Q10 metabolism has also been linked to this disorder. Occasionally, Leigh syndrome can occur spontaneously, termed "de novo."

Symptoms of Leigh syndrome typically advance rapidly. Early signs may manifest as poor sucking ability, loss of head control and motor skills, reduced appetite, vomiting, and seizures. As the disease progresses, additional symptoms such as muscle weakness, decreased muscle tone, spasticity, movement disorders, cerebellar ataxia, and peripheral neuropathy may develop. Complications can affect respiratory, cardiac, and renal function, contributing to further impairment.

Leigh Syndrome Treatment Market
The diagnosis of Leigh syndrome typically involves a comprehensive clinical assessment and specialized tests, particularly advanced imaging techniques. Magnetic resonance imaging (MRI) or computed tomography (CT) scans of the brain can reveal abnormal areas in specific brain regions such as the basal ganglia, brain stem, and gray matter.

In some cases, cysts of varying sizes may be detected in the cerebral cortex. Laboratory tests may show elevated levels of acidic waste products in the blood (lactic acidosis), as well as increased levels of pyruvate and alanine. Blood glucose levels may be slightly lower than normal. Additionally, deficiencies in enzymes like pyruvate carboxylase or cytochrome C oxidase may be present, and inhibitors affecting thiamine triphosphate (TTP) production may be found in blood and urine samples of affected individuals.

Currently, there is no specific treatment for Leigh syndrome, and therapies targeted specifically at mitochondrial disorders in children are generally limited. Symptomatic treatment focuses on managing symptoms and optimizing energy production by enhancing ATP levels and reducing lactate accumulation. This approach involves the elimination of harmful metabolites and the administration of oxygen radical scavengers. Antioxidants are commonly used in treatment. Symptomatic therapies for Leigh syndrome may also include supplementation with B vitamins such as thiamine, antiseizure medications, physiotherapy, and pharmacotherapy.

Nutritional supplements known as "nutraceuticals," derived from regular food sources, have been proposed as potential treatments for Leigh syndrome and other mitochondrial disorders. These include substances like coenzyme Q10, L-carnitine, alpha-lipoic acid, creatine monohydrate, biotin, thiamine, riboflavin, among others. Patients are often treated with combinations of these compounds in an attempt to manage symptoms and potentially improve outcomes.

Leigh Syndrome Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. 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 a detailed analysis of the diagnosed patient pool and future trends.
During the analysis of Leigh syndrome, it was determined that the prevalence of the condition is approximately 1 in 30,000 to 1 in 40,000 individuals at birth.
During the analysis, it was noted that 64% of Leigh syndrome patients exhibited a mutation in nuclear DNA. Among nuclear DNA genes, SURF1 was found to be the most prevalent, present in 72% of cases, among a total of 18 different nuclear DNA genes identified during the analysis.
During the analysis, the prevalence of Leigh Syndrome was determined to be 1 in 5,000 children in Japan.
Based on secondary research classical form of Leigh syndrome accounts for nearly 80% of cases.
The epidemiology of Leigh syndrome is expected to change during the forecast period (2024-2034).
Leigh Syndrome Epidemiology Segmentation:
The Leigh Syndrome market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Total Prevalence of Leigh Syndrome
Prevalent Cases of Leigh Syndrome by severity
Gender-specific Prevalence of Leigh Syndrome
Diagnosed Cases of Episodic and Chronic Leigh Syndrome

Download the report to understand which factors are driving Leigh Syndrome epidemiology trends @ Leigh Syndrome Epidemiology Forecast

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 during the study period. The analysis covers Leigh Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on 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 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.
Leigh Syndrome Emerging Drugs
NV354: Abliva AB

Discover more about therapies set to grab major Leigh Syndrome market share @ Leigh Syndrome Treatment Landscape

Scope of the Leigh Syndrome Market Report
Study Period: 2020–2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Leigh Syndrome Companies: NV354, and others
Key Leigh Syndrome Therapies: Abliva AB, and others
Leigh Syndrome Therapeutic Assessment: Leigh Syndrome current marketed and Leigh Syndrome emerging therapies
Leigh Syndrome Market Dynamics: Leigh Syndrome market drivers and Leigh Syndrome market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
Leigh Syndrome Unmet Needs, KOL’s views, Analyst’s views, Leigh Syndrome Market Access and Reimbursement

Table of Contents

1. Leigh Syndrome Market Report Introduction
2. Executive Summary for Leigh Syndrome
3. SWOT analysis of Leigh Syndrome
4. Leigh Syndrome Patient Share (%) Overview at a Glance
5. Leigh Syndrome Market Overview at a Glance
6. Leigh Syndrome Disease Background and Overview
7. Leigh Syndrome Epidemiology and Patient Population
8. Country-Specific Patient Population of Leigh Syndrome
9. Leigh Syndrome Current Treatment and Medical Practices
10. Leigh Syndrome Unmet Needs
11. Leigh Syndrome Emerging Therapies
12. Leigh Syndrome Market Outlook
13. Country-Wise Leigh Syndrome Market Analysis (2020–2034)
14. Leigh Syndrome Market Access and Reimbursement of Therapies
15. Leigh Syndrome Market Drivers
16. Leigh Syndrome Market Barriers
17. Leigh Syndrome Appendix
18. Leigh Syndrome Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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DelveInsight's 'Leigh Syndrome Epidemiology Forecast to 2034' report delivers an in-depth understanding of the disease, historical and forecasted Leigh Syndrome epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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About DelveInsight

DelveInsight is a leading Healthcare 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.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

Kritika Rehani
DelveInsight Business Research LLP
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