MELAS Syndrome Market

• According to DelveInsight, MELAS Syndrome market is expected to grow at a decent CAGR by 2032.
• 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.
• The disease epidemiology covered in the report provides historical as well as forecasted MELAS syndrome epidemiology [segmented as Total Prevalence of MELAS Syndrome, Gender-specific Prevalent cases of MELAS Syndrome, Mutation-specific Prevalent cases of MELAS Syndrome, and Diagnosed and treatable cases of MELAS Syndrome in the 7MM covering the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2019 to 2032.

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DelveInsight's "MELAS Syndrome Market Insights, Epidemiology, and Market Forecast 2032" report delivers an in-depth understanding of the MELAS syndrome, historical and forecasted epidemiology as well as the MELAS syndrome therapeutics market trends in the United States, EU4 (Germany, Spain, Italy, France) and the United Kingdom, and Japan.

The MELAS syndrome market report provides current treatment practices, emerging drugs, MELAS syndrome market share of the individual therapies, current and forecasted MELAS syndrome market Size from 2019 to 2032 segmented by seven major markets. The Report also covers current MELAS syndrome treatment practice/algorithm, and MELAS syndrome unmet medical needs to curate best of the opportunities and assesses the underlying potential of the market.

MELAS Syndrome Treatment Market

The DelveInsight’s MELAS syndrome market report gives a thorough understanding of MELAS syndrome by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis, and treatment. 

MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) is a mitochondrial disease primarily affecting the nervous system and muscles. MELAS presents in children or young adults as recurrent episodes of encephalopathy, myopathy, headache, and focal neurological deficits.

MELAS is a mitochondrial inherited genetic disorder. Mutations affecting the genes for mtDNA are inherited from the mother. MtDNA that is found in sperm cells is typically lost during fertilization, and as a result, all human mtDNA comes from the mother. An affected mother will pass on the mutation to all her children, but only her daughters will pass on the mutation to their children.

MELAS Syndrome Diagnosis

MELAS is diagnosed based on clinical findings and molecular genetic testing. Clinical testing includes measuring lactate, pyruvate, and CSF protein concentrations that are elevated in MELAS syndrome. Brain imaging techniques such as magnetic resonance imaging (MRI) can be used to look for stroke-like lesions, and magnetic resonance spectroscopy (MRS) may be used to look for a lactate peak in the brain. An electrocardiogram is used to diagnose heart rhythm abnormalities, and an echocardiogram may be used to diagnose cardiomyopathy. 

MELAS Syndrome Treatment

No specific treatment is available for MELAS syndrome. Anti-convulsant drugs are used to help prevent and control seizures associated with the syndrome.

MELAS Syndrome Epidemiology 

The MELAS syndrome epidemiology section provides insights about historical and current MELAS syndrome patient pool and forecasted trends for individual seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the MELAS syndrome market report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

• 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.
• The disease epidemiology covered in the report provides historical as well as forecasted MELAS syndrome epidemiology [segmented as Total Prevalence of MELAS Syndrome, Gender-specific Prevalent cases of MELAS Syndrome, Mutation-specific Prevalent cases of MELAS Syndrome, and Diagnosed and treatable cases of MELAS Syndrome in the 7MM covering the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2019 to 2032.
• A study conducted in Northern England determined that the prevalence of the m.3243A>G variant in the adult population was approximately 1 in 13,000.
• A study estimated that the prevalence of MELAS Syndrome among the general adult population in Spain is about 5.7 cases per 100,000 individuals.
• In 2012, the prevalence of MELAS syndrome was found to be approximately 0.18/100,000 in the total population of Japan.
• A comprehensive study in 2006 revealed a prevalence of the MELAS m.3243A>G variation to be 236/100,000, significantly higher than previously reported estimates.

Country Wise- MELAS Syndrome Epidemiology

This section provides glimpse of the MELAS syndrome epidemiology in the United States, EU4 (Germany, Spain, Italy, France) and United Kingdom, and Japan.

MELAS Syndrome Drug Chapters

The drug chapter segment of the MELAS syndrome treatment market report encloses the detailed analysis of MELAS syndrome marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the MELAS syndrome clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Marketed MELAS Syndrome Drugs

Note: Detailed Current therapies assessment will be provided in the full report of MELAS syndrome...

Emerging MELAS Syndrome Drugs

Sonlicromanol: Khondrion BV

Khondrion’s lead investigational asset, sonlicromanol (formerly known as KH176), stands out as a potential first-in-class medicine and represents one of the most advanced disease-modifying candidates for primary mitochondrial disease currently in clinical development. This innovative therapy is designed to address the fundamental mechanisms underlying mitochondrial disease through its unique triple mode of action, which includes modulation of both reductive and oxidative stress as well as anti-inflammatory effects.

Sonlicromanol has received Orphan Drug Designation for MELAS in Europe and all inherited mitochondrial respiratory chain disorders in the US. Additionally, it has been awarded the Rare Pediatric Disease designation by the US FDA for MELAS.

Currently, the drug is in Phase III of clinical development for the treatment of MELAS syndrome.

Zagociguat: Tisento Therapeutics

Zagociguat is an oral, once-daily investigational medication under development for mitochondrial diseases, starting with MELAS syndrome. As a clinical-stage sGC stimulator with CNS-penetrant properties, Zagociguat has demonstrated promising results in clinical studies, including rapid improvements in cerebral blood flow, functional brain connectivity, and cognitive performance. Additionally, the drug has positively influenced biomarkers related to mitochondrial function and inflammation.

Zagociguat is currently progressing through the Phase IIb PRIZM study for the treatment of MELAS syndrome.

KL1333: Abliva AB

KL1333 is being developed as a treatment for a subset of adult primary mitochondrial disease patients who experience multiple debilitating symptoms, including chronic fatigue and myopathy. The compound functions as a potent modulator of NAD⁺ and NADH, essential co-enzymes involved in cellular energy metabolism. In a Phase Ia/b study, patients receiving KL1333 demonstrated notable improvements in fatigue and functional status. KL1333 is now under evaluation in a global, potentially registrational Phase II study, known as the FALCON study, and has been granted orphan drug designation in both the US and Europe, along with fast-track designation in the US.

CY6463 (Cyclerion therapeutics) has completed two Phase I studies on healthy and old (>65 years) volunteers confirming targeting CNS exposure and activity. The status of the study as of May 2021 represents an ongoing phase of development and does not correspond to any particular phase of completion.

KL1333 (Abliva AB) has been evaluated in healthy volunteers and patients and has been granted Orphan Drug designation in both the United States and Europe. Abliva plans to recruit the first patient in a registrational Phase II/III study in 2022. KL1333 is a potent modulator of the cellular levels of NAD+, a central coenzyme in the cell’s energy metabolism.

Note: Detailed emerging therapies assessment will be provided in the full report of MELAS syndrome 

MELAS Syndrome Market Outlook

Anticonvulsant medications are often used to prevent seizures that can occur with MELAS. The symptomatic treatment of seizures is done with anti-epileptic medications. There are several case reports of aggravation of MELAS epilepsy with the use of valproate, though the mechanisms behind this interaction are still being investigated. However, they are unable to confirm the condition via genetic diagnosis. Such instances are further comlicated by the aforementioned uncertainty, leading to delays in treatment and subsequent deterioration in the patient’s quality of life.

L-arginine has been shown to attenuate the severity of symptoms when used in acute attacks and decrease the frequency of episodes. L-citrulline is also believed to be beneficial in the recovery reduction of stroke risk. This relationship is theorized to be due to the correction of nitric oxide deficiency in MELAS patients, as arginine and citrulline are precursors to nitric oxide production.

Key MELAS syndrome companies such as Cyclerion therapeutics, Abliva AB, Khondrion along with few others are involved in developing therapies for MELAS syndrome.

According to DelveInsight, MELAS syndrome market in 7MM is expected to witness a major change in the study period 2019-2032.

Key findings

This section includes a glimpse of the MELAS syndrome market in 7MM.

The United States: MELAS Syndrome Market Outlook

This section provides the total MELAS syndrome market size and market size by therapies in the United States.

EU4 and the UK: MELAS Syndrome Market Outlook

The total MELAS syndrome market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Japan: MELAS Syndrome Market Outlook

The total MELAS syndrome market size and market size by therapies in Japan is also mentioned.

Analyst Commentary

• The pipeline of MELAS syndrome is very robust, many potential therapies are being investigated for the treatment of MELAS syndrome, and it is safe to predict that the treatment space will experience a significant impact on the MELAS syndrome market during the forecast period.
• The expected introduction of emerging MELAS syndrome therapies with improved efficacy, more awareness initiatives programs, and further improvement in the diagnosis rate, are likely to boost the growth of the MELAS syndrome market in the 7MM. Aside from that, the market size of MELAS syndrome may flourish due to increased research and development, label-expansion of approved therapies into other epilepsy in this field.
• The MELAS syndrome market growth omay offset by failures and/or discontinuation of the emerging therapies, unaffordable pricing, market access and reimbursement issues, and a scarcity of healthcare specialists.

MELAS Syndrome Drugs Uptake

This section focusses on the rate of uptake of the potential MELAS syndrome drugs recently launched in the MELAS syndrome market or expected to get launched in the MELAS syndrome drugs market during the study period 2019-2032. The analysis covers MELAS syndrome market uptake by drugs; patient uptake by therapies; and sales of each drug.

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

Note: Detailed emerging therapies assessment will be provided in full report of MELAS syndrome.

MELAS Syndrome Pipeline Development Activities

The report provides insights into MELAS syndrome clinical trials within Phase II and Phase III stage. It also analyses key MELAS syndrome companies involved in developing targeted therapeutics.

MELAS Syndrome Clinical Trials Development Activities

The MELAS Syndrome clinical trials market report covers the detailed information of collaborations, acquisition and merger, licensing, patent details and other information for emerging MELAS syndrome therapies.

Reimbursement Scenario in MELAS Syndrome 

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In a report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

KOL- Views on MELAS syndrome

To keep up with current MELAS syndrome market trends, we take KOLs and SMEs ' opinions working in the MELAS syndrome domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or MELAS syndrome market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the MELAS syndrome unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the MELAS syndrome Market by using various Competitive Intelligence tools that include - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.

MELAS Syndrome Market Report Scope

• The MELAS syndrome market report covers the descriptive overview of MELAS  syndrome, explaining its causes, signs and symptoms, pathophysiology, diagnosis, and currently available MELAS syndrome therapies
• Comprehensive insight has been provided into the MELAS syndrome epidemiology and treatment in the 7MM
• Additionally, an all-inclusive account of both the current and emerging therapies for MELAS syndrome are provided, along with the assessment of new therapies, which will have an impact on the current MELAS syndrome treatment landscape
• A detailed review of the MELAS syndrome market; historical and forecasted is included in the report, covering drug outreach in the 7MM
• The patient-based MELAS syndrome market forecasting report provides an edge while developing business strategies, by understanding trends shaping and driving the global MELAS syndrome market

MELAS Syndrome Market Report Highlights

• In the coming years, the MELAS syndrome market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the MELAS syndrome drug manufacturers to penetrate more into the MELAS syndrome market
• The MELAS syndrome companies and academics are working to assess challenges and seek opportunities that could influence MELAS syndrome R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition      
• Major MELAS syndrome companies are involved in developing therapies for MELAS syndrome. The launch of emerging therapies will significantly impact the MELAS syndrome treatment market
• A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for MELAS syndrome 
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

MELAS Syndrome Market Report Insights

• Patient-Based MELAS syndrome Market Forecasting
• MELAS syndrome Therapeutic Approaches
• MELAS Syndrome Pipeline Analysis
• MELAS Syndrome Market Size
• MELAS syndrome Market Trends
• MELAS syndrome Market Opportunities
• Impact of upcoming MELAS syndrome Therapies

MELAS Syndrome Market Report Key Strengths

• 11 Years Forecast
• 7MM Coverage
• MELAS Syndrome Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed MELAS syndrome Market
• MELAS syndrome Drugs Uptake

MELAS Syndrome Market Report Assessment

• Current MELAS syndrome Treatment Practices
• MELAS syndrome Unmet Needs
• MELAS syndrome Pipeline Product Profiles
• MELAS syndrome Market Attractiveness
• MELAS syndrome Market Drivers
• MELAS syndrome Market Barriers

Key Questions Answered In The MELAS syndrome Market Report:

MELAS Syndrome Market Insights:

• What was the MELAS syndrome drug class share (%) distribution in 2019 and how it would look like in 2032?
• What would be the MELAS syndrome market size as well as market size by therapies across the 7MM during the forecast period (2019-2032)?
• What are the key findings of the market across 7MM and which country will have the largest MELAS syndrome market size during the forecast period (2019-2032)?
• At what CAGR, the MELAS syndrome market is expected to grow by 7MM during the forecast period (2019-2032)?
• What would be the MELAS syndrome market outlook across the 7MM during the forecast period (2019-2032)?
• What would be the MELAS syndrome market growth till 2032, and what will be the resultant market Size in the year 2032?
• How would the unmet needs affect the MELAS syndrome market dynamics and subsequent analysis of the associated trends?

MELAS syndrome Epidemiology Insights:

• What are the disease risk, burden, and regional/ethnic differences of MELAS syndrome?
• What are the key factors driving the epidemiology trend for seven major markets covering the United States, EU4 (Germany, Spain, France, Italy) and the UK, and Japan?
• What is the historical MELAS syndrome patient pool in seven major markets covering the United States, EU4 (Germany, Spain, France, Italy) and the UK, and Japan?
• What would be the forecasted patient pool of MELAS syndrome in seven major markets covering the United States, EU4 (Germany, Spain, France, Italy) and the UK, and Japan?
• Where will be the growth opportunities in the 7MM concerning the patient population about MELAS syndrome?
• Out of all 7MM countries, which country would have the highest prevalence population of MELAS syndrome during the forecast period (2019-2032)?
• At what CAGR the patient population is expected to grow by 7MM during the forecast period (2019-2032)?

Current MELAS syndrome Treatment Scenario, Marketed Drugs, and Emerging Therapies:

• What are the current options for the MELAS syndrome treatment in addition to the approved therapies?
• What are the current treatment guidelines for the treatment of MELAS syndrome in the USA, Europe, and Japan?
• What are the MELAS syndrome marketed drugs and their respective MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, efficacy, etc.?
• How many MELAS syndrome companies are developing therapies for the treatment of MELAS syndrome?
• How many MELAS syndrome therapies are in-development by each company for MELAS syndrome treatment?
• How many are emerging MELAS syndrome therapies in mid-stage, and late stage of development for MELAS syndrome treatment?
• What are the key collaborations (Industry - Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the MELAS syndrome therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for MELAS syndrome and its status?
• What are the current challenges faced in MELAS syndrome drug development?
• What are the key designations that have been granted for the emerging therapies for MELAS syndrome?
• What are the global historical and forecasted MELAS syndrome market

Reasons to buy MELAS syndrome Market Forecast Report

• The patient-based MELAS syndrome market forecasting report will help in developing business strategies by understanding trends shaping and driving the MELAS syndrome market
• Organize sales and marketing efforts by identifying the best opportunities for MELAS syndrome in the US, Europe (Germany, Spain, Italy, France and the United Kingdom), and Japan
• Identification of strong upcoming MELAS syndrome companies in the market will help in devising strategies that will help in getting ahead of competitors
• Organize sales and marketing efforts by identifying the best opportunities for the MELAS syndrome market
• To understand the future market competition in the MELAS syndrome market

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