MELAS Syndrome Market is Predicted to Exhibit Remarkable Growth During the Forecast Period (2022-2032), Analyzes DelveInsight | Key Companies - Cyclerion Therapeutics (zagociguat/CY6463), Abliva AB (KL1333), Khondrion (Sonlicromanol)

As per DelveInsight, the MELAS Syndrome Market is anticipated to evolve immensely in the coming years owing to the increase in the prevalence of MELAS Syndrome and the expected launch of emerging therapies in the market. Several major pharma and biotech companies across the globe are diligently working toward developing novel treatment therapies with a considerable amount of success over the years. The leading Companies in the domain include Cyclerion therapeutics, Abliva AB, and Khondrion.

DelveInsight's "MELAS 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 MELAS Syndrome Market Size and Share in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).

The MELAS Syndrome market report covers emerging drugs, treatment practices, market share of individual therapies, and current & 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 opportunities and assess the underlying potential of the market.

MELAS Syndrome: An Overview
MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes) syndrome is a rare disorder that begins in childhood, usually between two and fifteen years of age, and mostly affects the nervous system and muscles. The most common early symptoms are seizures, recurrent headaches, loss of appetite, and recurrent vomiting.

Symptoms of MELAS syndrome usually begin between the ages of two and fifteen years, but delayed onset cases have also been reported between fifteen and forty years, and late-onset cases after forty years. MELAS is caused by mutations in mitochondrial DNA. Some cases of MELAS syndrome appear to occur as the result of a new spontaneous mutation in a mitochondrial gene and are not inherited. It affects males and females in equal numbers.

MELAS Syndrome Market Key Facts
• According to Pia et al. (2021), MELAS is one of the most common mitochondrial diseases, with an estimated incidence of 1 in 4000. Both genders are equally affected, but only women can pass the condition on, as mitochondria are carried in the tails of sperm cells and therefore shed outside the zygote during fertilization.
• As per Yatsuga et al. (2012), the prevalence of MELAS was estimated to be 0.2/100,000 in Japan.
• According to a study by Uusimaa et al. (2007), the prevalence of 3243A>G is relatively high in the pediatric population, but the morbidity in children is relatively low. The random genetic drift model may be inappropriate for transmitting the 3243A>G mutation.

Major Clinical, Commercial, and Regulatory Developments in the MELAS Syndrome Therapeutics Market
• On March 27, 2023, Cyclerion Therapeutics, Inc. (Nasdaq: CYCN) announced that the U.S. Food and Drug Administration (FDA) had granted orphan drug designation to zagociguat (previously CY6463) for the treatment of mitochondrial diseases. Zagociguat is the first CNS-penetrant sGC stimulator to be developed as a symptomatic and potentially disease-modifying therapy for serious diseases that involve the CNS.
• On 22 November 2022, Khondrion provides an update from (A) the 28-day double-blind, randomized, placebo-controlled, three-way cross-over KHENERGYZE Phase IIb study in 27 patients, and (B) the ongoing, open-label extension KHENEREXT Phase IIb study (7 patients up to 52 weeks) (both studies jointly the "Phase IIb programme"). Sonlicromanol, Khondrion's wholly-owned lead asset, is being investigated in the Phase IIb program in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
• On September 28, 2020, Khondrion announced that it received a rare pediatric disease (RPD) designation from the United States (US) Food and Drug Administration (FDA) for sonlicromonal for the treatment of patients with MELAS syndrome.

MELAS Syndrome Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted MELAS 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 complete detail of the MELAS 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.

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MELAS Syndrome Epidemiology
The epidemiology section covers detailed insights into the historical and current MELAS Syndrome patient pool and forecasted trends for every seven major countries (7MM) from 2019 to 2032.

The Report Covers the MELAS Syndrome Epidemiology Segmentation as follows -
• Total Prevalence of MELAS Syndrome in the 7MM [2019-2032]
• Diagnosed and treatable cases of MELAS Syndrome in the 7MM [2019-2032]
• Gender-specific Prevalent cases of MELAS Syndrome in the 7MM [2019-2032]
• Mutation-specific Prevalent cases of MELAS Syndrome in the 7MM [2019-2032]

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MELAS 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 MELAS Syndrome market or expected to be launched during the study period. The analysis covers MELAS Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug.

The report's drugs uptake section helps in understanding the drugs with the most rapid uptake and reasons behind the maximal use of new drugs and allows the comparison of the drugs based on 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 MELAS 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 recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

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MELAS Syndrome Therapeutics Assessment
MELAS is a genetically defined orphan disease for which there is no approved treatment. The defining features of MELAS are stroke-like episodes and lactic acidosis, a buildup of lactic acid in the bloodstream. Common CNS symptoms include seizures, headaches, migraines, and cognitive impairment.

To improve the treatment scenario, several major pharma and biotech companies are developing therapies for MELAS Syndrome. Several emerging therapies are awaiting approval. Some emerging therapies are in the mid and late phases of clinical trials. Currently, Cyclerion Therapeutics Inc. is leading the therapeutics market with its MELAS Syndrome drug candidates in the most advanced stage of clinical development.

The Leading Companies in the MELAS Syndrome Therapeutics Market Include:
• Cyclerion Therapeutics Inc.
• Khondrion BV
• Abliva AB
And Many Others

MELAS Syndrome Emerging and Marketed Drugs Covered in the Report Include:
• CY6463 (Cyclerion therapeutics)
• KL1333 (Abliva AB)
• Sonlicromanol (Khondrion)
And Many More

Learn More About the Emerging Therapies & Key Companies in the MELAS Syndrome Therapeutics Market:
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. MELAS Syndrome Competitive Intelligence Analysis
4. MELAS Syndrome Market Overview at a Glance
5. MELAS Syndrome Background and Overview
6. MELAS Syndrome Patient Journey
7. MELAS Syndrome Epidemiology and Patient Population
8. MELAS Syndrome Treatment Algorithm, Current Treatment, and Medical Practices
9. MELAS Syndrome Unmet Needs
10. Key Endpoints of MELAS Syndrome Treatment
11. MELAS Syndrome Marketed Products
12. MELAS Syndrome Emerging Therapies
13. MELAS Syndrome Seven Major Market Analysis
14. Attribute Analysis
15. MELAS Syndrome Market Outlook (7 major markets)
16. MELAS Syndrome Access and Reimbursement Overview
17. KOL Views on the MELAS Syndrome Market.
18. MELAS Syndrome Market Drivers
19. MELAS 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
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