Giant Axonal Neuropathy Market

 

Key Highlights

 

• According to Opal (2021), Giant axonal neuropathy is a very rare disorder. To date, approximately 50 families have been identified with biallelic GAN pathogenic variants. The condition is thought to be underdiagnosed because its early symptoms resemble those of other, more common disorders affecting the peripheral nervous system, such as Charcot-Marie-Tooth disease. Also, as per the data availability in European Organisation for Rare Diseases (EURODIS) also, the disease affects a very small number of children. Currently, there are probably less than 50 confirmed cases of GAN in the whole world.

 

• The Giant Axonal Neuropathy epidemiology covered in the report provides historical as well as forecasted Giant Axonal Neuropathy epidemiology [segmented as Total Prevalent Cases of Giant Axonal Neuropathy, Total Diagnosed Prevalent Cases of Giant Axonal Neuropathy, and Treated Cases of Giant Axonal Neuropathy] in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2019 to 2032.

 

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DelveInsight’s “Giant Axonal Neuropathy – Market Insights, Epidemiology, and Market Forecast – 2032” report delivers an in-depth understanding of the Giant Axonal Neuropathy, historical and forecasted epidemiology as well as the Giant Axonal Neuropathy market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

 

The Giant Axonal Neuropathy market report provides current treatment practices, Giant Axonal Neuropathy emerging therapies, the Giant Axonal Neuropathy market share of individual therapies, and current and forecasted Giant Axonal Neuropathy market size from 2019 to 2032, segmented by the seven major markets. The report also covers current Giant Axonal Neuropathy treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

 

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

 

Study period: 2019–2032

 

Giant Axonal Neuropathy Market: Understanding and Treatment Algorithm

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

 

Giant axonal neuropathy (GAN) is a pediatric, hereditary, neurodegenerative disorder that affects both the central and peripheral nervous systems. The peripheral nervous system sends signals that control movement and sensation between the central nervous system (the brain and spinal cord) and all other parts of the body. 

 

It is caused by mutations in the GAN gene, which codes for the gigaxonin protein. Gigaxonin plays a role in intermediate filament (IF) turnover hence loss of function of this protein leads to IF aggregates in various types of cells. These aggregates can lead to abnormal cellular function that manifests as a diverse set of symptoms in persons with GAN including nerve degeneration, cognitive issues, skin diseases, vision loss, and muscle weakness. GAN happens when the GAN1 gene is changed and causes the axons, or the message sender of the nerve cell, to become larger than normal and not function properly. After some time, these axons break down and affect movement and feeling because the nerve cells can't talk to each other.

 

GAN is progressive, meaning that it continues to worsen over time. Most children begin showing signs of GAN before five years of age. Most children need to use a wheelchair in the second decade of life, and some may survive into early adulthood. Currently, there is no cure or treatment to stop the disorder from progressing.

 

Early signs include difficulty walking, lack of coordination, and loss of strength. Over time, the central nervous system (brain and spinal cord) becomes involved, causing a gradual decline in mental function, loss of control of body movements, and seizures. The disease follows an autosomal dominant pattern of inheritance.

 

Giant Axonal Neuropathy Diagnosis

Doctors diagnose GAN by using several tests, including one that measures nerve conduction velocity, a brain MRI, and a peripheral nerve biopsy (in which a bit of tissue from a peripheral nerve is removed and examined to look for swollen axons).  The hallmark finding on a peripheral nerve biopsy is the appearance of "giant axons" which are caused by the accumulation of neurofilaments. Prenatal diagnosis is possible in families with a known disease-causing mutation.

 

Giant Axonal Neuropathy Treatment

Presently there is no effective treatment for GAN. Only symptomatic management to slow the progression of the disease are in place. A combination of speech, occupation, and physical therapy is used by physicians to enhance physical and cognitive development. As such, there is a need for a therapy that provides an effective yet safe treatment that addresses the primary cause of GAN.

 

Treatment is symptomatic. Children with GAN and their families usually work with a medical team that includes a pediatric neurologist, orthopedic surgeon, physiotherapist, psychologist, and speech and occupational therapists. The major goals of treatment are to maximize intellectual and physical development and minimize their deterioration as time passes.  Many children with GAN begin with normal intellectual development and can attend a regular school program. Children should be monitored at least once a year to assess their intellectual abilities and to look for the presence of neurological deterioration.

 

With remarkable advances made in the molecular biology field, several strategies to fight GAN are evolving. Although there is no cure for GAN at the moment, with the ongoing Giant Axonal Neuropathy clinical trials and several other available strategies to treat neurological maladies, patients and their families can be hopeful.

 

Giant Axonal Neuropathy Epidemiology 

The Giant Axonal Neuropathy epidemiology section provides insights into the historical and current Giant Axonal Neuropathy patient pool and forecasted trends for the seven individual major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Giant Axonal Neuropathy report also provides the prevalent patient pool, its trends, and assumptions undertaken.

 

Key findings

 

According to Opal (2021), Giant axonal neuropathy is a very rare disorder. To date, approximately 50 families have been identified with biallelic GAN pathogenic variants. The condition is thought to be underdiagnosed because its early symptoms resemble those of other, more common disorders affecting the peripheral nervous system, such as Charcot-Marie-Tooth disease. Also, as per the data availability in European Organisation for Rare Diseases (EURODIS) also, the disease affects a very small number of children. Currently, there are probably less than 50 confirmed cases of GAN in the whole world.

 

The Giant Axonal Neuropathy epidemiology covered in the report provides historical as well as forecasted Giant Axonal Neuropathy epidemiology [segmented as Total Prevalent Cases of Giant Axonal Neuropathy, Total Diagnosed Prevalent Cases of Giant Axonal Neuropathy, and Treated Cases of Giant Axonal Neuropathy] in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2019 to 2032.

 

Country-wise Giant Axonal Neuropathy Epidemiology

The epidemiology segment also provides the Giant Axonal Neuropathy epidemiology data and findings across the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

 

Giant Axonal Neuropathy Drug Chapters

The drug chapter segment of the Giant Axonal Neuropathy report encloses a detailed analysis of Giant Axonal Neuropathy marketed drugs and late-stage (Phase III and Phase II) Giant Axonal Neuropathy pipeline drugs. It also helps understand the Giant Axonal Neuropathy 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.

 

Emerging Giant Axonal Neuropathy Drugs

The report details the emerging Giant Axonal Neuropathy therapies under the late and mid-stage of development for Giant Axonal Neuropathy treatment.

 

Giant Axonal Neuropathy Market Outlook

The Giant Axonal Neuropathy market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Giant Axonal Neuropathy market trends by analyzing the impact of current therapies on the market, unmet needs, and demand for better technology.

 

This segment gives a thorough detail of the Giant Axonal Neuropathy market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need for the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

 

According to DelveInsight, the Giant Axonal Neuropathy market in the 7MM is expected to witness a major change in the study period 2019–2032.

 

Key findings

This section includes a glimpse of the Giant Axonal Neuropathy market in the 7MM.

 

The United States: Giant Axonal Neuropathy Market Outlook

This section provides the total Giant Axonal Neuropathy market size and market size by therapies in the United States.

 

EU4 and the United Kingdom: Giant Axonal Neuropathy Market Outlook

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

 

Japan: Giant Axonal Neuropathy Market Outlook

The total Giant Axonal Neuropathy market size and market size by therapies in Japan are also mentioned.

 

Giant Axonal Neuropathy Drug Uptake

This section focuses on the rate of uptake of the potential drugs recently launched in the Giant Axonal Neuropathy market or expected to get launched in the market during the study period 2019–2032. The analysis covers the Giant Axonal Neuropathy market uptake by drugs, patient uptake by therapies, and sales of each drug.  

 

This will help in understanding the Giant Axonal Neuropathy drugs with the most rapid uptake and the 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 the market uptake and in making financial and regulatory decisions.

 

Giant Axonal Neuropathy Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses Giant Axonal Neuropathy’s key players involved in developing targeted therapeutics.

 

Giant Axonal Neuropathy Development Activities

The report covers detailed information on collaborations, acquisitions, and mergers, licensing patent details, and other information on Giant Axonal Neuropathy emerging therapies.

 

Giant Axonal Neuropathy Reimbursement Scenario

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

To keep up with current epidemiology and market trends, we take KOLs and SMEs' opinions working on the Giant Axonal Neuropathy 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 and treatment patterns along with Giant Axonal Neuropathy market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

 

Competitive Intelligence Analysis

We perform competitive and market intelligence analysis of the Giant Axonal Neuropathy 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.

 

Scope of the Giant Axonal Neuropathy Market Report

• Descriptive overview of Giant Axonal Neuropathy, disease overview, patient journeys, treatment algorithms, diagnosis, and currently available therapies
• Comprehensive insight into the Giant Axonal Neuropathy epidemiology and forecasts in the 7MM
• An all-inclusive account of both the current and emerging therapies for Giant Axonal Neuropathy, along with the assessment of new therapies, expected to have an impact on the current treatment landscape
• Exhaustive analysis of the Giant Axonal Neuropathy market; historical and forecasted covering drug outreach in the 7MM
• Detailed patient-based Giant Axonal Neuropathy market forecasting determines the trends shaping and driving the global Giant Axonal Neuropathy market

 

Giant Axonal Neuropathy Market Report Highlights

• In the coming years, the Giant Axonal Neuropathy 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 drug manufacturers to penetrate more into the market
• The Giant Axonal Neuropathy companies and academics are working to assess challenges and seek opportunities that could influence Giant Axonal Neuropathy R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition   
• Major players are involved in developing Giant Axonal Neuropathy therapies. The launch of emerging therapies will significantly impact the Giant Axonal Neuropathy market
• A better understanding of Giant Axonal Neuropathy pathogenesis will also contribute to the development of novel therapeutics for Giant Axonal Neuropathy
• Our in-depth analysis of the Giant Axonal Neuropathy pipeline assets across different stages of development (Phase III and Phase II), emerging trends, and comparative analysis of pipeline products with detailed Giant Axonal Neuropathy clinical trials 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

 

Giant Axonal Neuropathy Report Insights

• Patient-Based Giant Axonal Neuropathy Market Forecasting 
• Therapeutic approaches
• Giant Axonal Neuropathy pipeline analysis
• Giant Axonal Neuropathy market size and trends
• Giant Axonal Neuropathy market opportunities
• Impact of upcoming therapies

 

Giant Axonal Neuropathy Report Key Strengths

• 11 years forecast
• The 7MM Coverage
• Giant Axonal Neuropathy epidemiology segmentation
• Key cross competition
• KOL views
• Giant Axonal Neuropathy drugs uptake

 

Giant Axonal Neuropathy Report Assessment

• Current treatment practices
• Giant Axonal Neuropathy Unmet needs
• Giant Axonal Neuropathy pipeline product profiles
• Giant Axonal Neuropathy market attractiveness

 

Key Questions

Giant Axonal Neuropathy market insights:

• What would be the Giant Axonal Neuropathy market growth till 2032, and what will be the resultant market size in 2032?
• What was the Giant Axonal Neuropathy drug class share (in percentage) distribution in 2019, and how would it look in 2032?
• What would be the Giant Axonal Neuropathy total market size and market size by therapies across the 7MM during the forecast period (2022–2032)?
• What are the key findings of the market across the 7MM, and which country will have the largest Giant Axonal Neuropathy market size during the forecast period (2022–2032)?
• How would the unmet needs affect the Giant Axonal Neuropathy market dynamics and subsequent analysis of the associated trends?

 

Giant Axonal Neuropathy Epidemiology Insights:

• What are the disease risk, burden, and regional/ethnic differences of Giant Axonal Neuropathy?
• What is the historical and forecasted Giant Axonal Neuropathy patient pool in the 7MM, and where can one observe the highest patient population and growth opportunities? 
• What are the key factors driving the epidemiology trends for the seven major markets covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?

 

Current Treatment Scenario, Marketed Drugs, and Giant Axonal Neuropathy Emerging Therapies

• What are the current treatment guidelines and treatment options, in addition to approved therapies for Giant Axonal Neuropathy in the United States, Europe, and Japan?
• What are the key collaborations (Industry–Industry, Industry-Academia), mergers and acquisitions, and licensing activities related to Giant Axonal Neuropathy 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 Giant Axonal Neuropathy and its status, along with the challenges faced?

 

Reasons to Buy

·         The patient-based Giant Axonal Neuropathy market forecasting analysis will help in developing business strategies by understanding trends shaping and driving the Giant Axonal Neuropathy market

 

·         Organize sales and marketing efforts by identifying the best opportunities for Giant Axonal Neuropathy in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan

·         Identification of strong upcoming players in the market that will help devise strategies that will help in getting ahead of competitors