Giant Axonal Neuropathy Market: Analysis of Epidemiology, Pipeline Products, and Key Companies Working in the Market | Companies-Taysha Gene Therapies and others

DelveInsight has launched a new report on "Giant Axonal Neuropathy - Market Insights, Epidemiology, and Market Forecast-2032".
DelveInsight's "Giant Axonal Neuropathy (GAN)- Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Giant Axonal Neuropathy (GAN), historical and forecasted epidemiology as well as the Giant Axonal Neuropathy (GAN) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
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Some of the key facts of the Giant Axonal Neuropathy Market Report:
According to Opal (2021), Giant Axonal Neuropathy (GAN) is an extremely rare disorder, with approximately 50 families worldwide identified with biallelic GAN pathogenic variants to date. The condition is often underdiagnosed because its early symptoms closely resemble those of other, more common peripheral nervous system disorders, such as Charcot-Marie-Tooth disease. Data from the European Organisation for Rare Diseases (EURODIS) also indicates that GAN affects a very small number of children, with fewer than 50 confirmed cases globally.
The epidemiology section of the report provides both historical and forecasted data on Giant Axonal Neuropathy, including the total prevalent cases, diagnosed prevalent cases, and treated cases. This data covers the 7MM (United States, EU4 countries-Germany, France, Italy, and Spain-the United Kingdom, and Japan) from 2019 to 2032.
Key benefits of the Giant Axonal Neuropathy market report:
1. Giant Axonal Neuropathy market report covers a descriptive overview and comprehensive insight of the Giant Axonal Neuropathy Epidemiology and Giant Axonal Neuropathy market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan.)
2. The Giant Axonal Neuropathy market report provides insights into the current and emerging therapies.
3. Giant Axonal Neuropathy market report provides a global historical and forecasted market covering drug outreach in 7MM.
4. The Giant Axonal Neuropathy market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Giant Axonal Neuropathy market.
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Giant Axonal Neuropathy Overview
Giant Axonal Neuropathy (GAN) is a rare, hereditary neurodegenerative disorder that affects both the central and peripheral nervous systems. The peripheral nervous system transmits signals controlling movement and sensation between the central nervous system (the brain and spinal cord) and the rest of the body.

GAN is caused by mutations in the GAN gene, which encodes the gigaxonin protein. Gigaxonin is crucial for the turnover of intermediate filaments (IFs), and a loss of function of this protein leads to the accumulation of IF aggregates in various cell types. These aggregates disrupt normal cellular function, resulting in a range of symptoms such as nerve degeneration, cognitive impairments, skin disorders, vision loss, and muscle weakness. The disease occurs when mutations in the GAN1 gene cause axons-responsible for transmitting nerve signals-to become enlarged and dysfunctional. Over time, these axons deteriorate, impairing communication between nerve cells and affecting movement and sensation.

GAN is a progressive condition, meaning it worsens over time. Symptoms typically begin in children before the age of five, with most needing a wheelchair by their second decade of life. Some individuals may survive into early adulthood. Currently, there is no cure or treatment to halt the progression of the disorder.

Early symptoms include difficulty walking, lack of coordination, and muscle weakness. As the disease advances, it progressively affects the central nervous system, leading to a decline in cognitive function, loss of motor control, and seizures. GAN follows an autosomal dominant inheritance pattern.

Giant Axonal Neuropathy Diagnosis
Doctors diagnose Giant Axonal Neuropathy (GAN) using various tests, including nerve conduction velocity measurements, brain MRI, and peripheral nerve biopsy. During a peripheral nerve biopsy, a small sample of nerve tissue is removed and examined for swollen axons. The key diagnostic feature in a peripheral nerve biopsy is the presence of "giant axons," which result from the buildup of neurofilaments. In families with a known disease-causing mutation, prenatal diagnosis is also possible.
Giant Axonal Neuropathy Treatment
Currently, there is no cure for Giant Axonal Neuropathy (GAN), and treatment focuses on managing symptoms to slow disease progression. Physicians use a combination of speech, occupational, and physical therapy to improve physical and cognitive development. There is a pressing need for therapies that provide both effective and safe treatments targeting the root cause of GAN.

Treatment remains symptomatic, with a comprehensive medical team often involved, including a pediatric neurologist, orthopedic surgeon, physiotherapist, psychologist, and speech and occupational therapists. The primary goals are to maximize intellectual and physical development while minimizing deterioration over time. Many children with GAN initially develop normally and can participate in regular school programs. Regular monitoring, at least once a year, is essential to assess intellectual abilities and detect any neurological decline.

Significant advancements in molecular biology are leading to new strategies for addressing GAN. While no cure exists yet, ongoing clinical trials and emerging treatments for neurological disorders offer hope to patients and their families.
Giant Axonal Neuropathy Epidemiology
According to Opal (2021), Giant Axonal Neuropathy (GAN) is an extremely rare disorder, with around 50 families worldwide identified as having biallelic GAN pathogenic variants. The condition is likely underdiagnosed due to its early symptoms closely resembling those of more common peripheral nervous system disorders, such as Charcot-Marie-Tooth disease. Data from the European Organisation for Rare Diseases (EURODIS) confirms that GAN impacts a very limited number of children. Currently, there are estimated to be fewer than 50 confirmed cases of GAN globally.
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Giant Axonal Neuropathy Market
The dynamics of the Giant Axonal Neuropathy market are expected to shift in the coming years, driven by the anticipated introduction of new therapies from companies like Taysha Gene Therapies and others during the forecast period from 2019 to 2032.
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Giant Axonal Neuropathy Pipeline Therapies and Key Companies
• TSHA-120: Taysha Gene Therapies
And others.
Giant Axonal Neuropathy Market Drivers
• Active participation of the different organizations in increased R&D
• Regulatory incentives such as market exclusivity for orphan designated drugs
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Table of Contents
1. Report Introduction
2. Executive Summary
3. SWOT analysis
4. Giant Axonal Neuropathy Patient Share (%) Overview at a Glance
5. Giant Axonal Neuropathy Market Overview at a Glance
6. Giant Axonal Neuropathy Disease Background and Overview
7. Giant Axonal Neuropathy Epidemiology and Patient Population
8. Country-Specific Patient Population of Giant Axonal Neuropathy
9. Giant Axonal Neuropathy Current Treatment and Medical Practices
10. Unmet Needs
11. Giant Axonal Neuropathy Emerging Therapies
12. Giant Axonal Neuropathy Market Outlook
13. Country-Wise Giant Axonal Neuropathy Market Analysis (2019-2032)
14. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Giant Axonal Neuropathy Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
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