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Delveinsight Business Research LLP

The report covers emerging Giant Axonal Neuropathy drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Giant Axonal Neuropathy treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market. 

Giant Axonal Neuropathy: An Overview

Giant Axonal Neuropathy (GAN) is a rare inherited genetic disorder that severely affects the peripheral as well as the central nervous system. GAN is characterized by abnormally large and dysfunctional axons called giant axons. The onset of GAN ranges from early infancy to late childhood

This neurodegenerative disorder is caused by mutations in the GAN gene located on chromosome 16 at 16q24.1 which provides instructions for making a protein called gigaxonin protein. The nonstandard gigaxonin protein causes a portion of the nerve cell called the axon to swell up with deposits of tiny threads of a protein called neurofilaments, giving the presence of giant axons. The giant axons cause degeneration and abnormal functioning of the peripheral nervous system.

This condition is inherited in an autosomal recessive pattern, which means both copies of the gene present in each cell have mutations. The parents of an individual with an autosomal recessive condition each carry one copy of the mutated gene, but they usually do not show signs and symptoms of the condition. As per some studies, since the first GAN report in 1972, more than 50 GAN gene mutations have been identified.

Treatment of GAN is symptomatic and supportive in nature and often encompasses multiple teams of medical professionals for visually and/or mobility impaired people who may be of help to people with GAN.

Giant Axonal Neuropathy Market Key Facts

• As per the Orphanet (2021), the prevalence of GAN is unknown, but to date, approximately 50 families have been reported. The frequency of this disease, however, is likely to be underestimated, due to patients with incomplete phenotypes and high levels of consanguinity among some populations. It occurs equally in both sexes.

• According to NORD, GAN affects equal numbers of males and females. The prevalence of giant axonal neuropathy and the frequency of carriers of one defective copy of the GAN gene is not known, but it is known that giant axonal neuropathy is a very rare disease.

• As per the data by the European Organisation for Rare Diseases (EURODIS), the disease affects a very small number of children. Currently, there are probably less than 50 confirmed cases of GAN in the whole world.

Giant Axonal Neuropathy Market

The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Giant Axonal Neuropathy pipeline therapies. It also thoroughly assesses the Giant Axonal Neuropathy market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.

The report gives complete details of the market trend for each marketed Giant Axonal Neuropathy drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.

Giant Axonal Neuropathy Epidemiology Assessment 

The epidemiology section provides insights into the historical, current, and forecasted Giant Axonal Neuropathy epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Giant Axonal Neuropathy epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders. 

The Report Covers the Giant Axonal Neuropathy Epidemiology, Segmented as –

• Total Prevalent Cases of Giant Axonal Neuropathy

• Total Diagnosed Prevalent Cases of Giant Axonal Neuropathy

• Treated Cases of Giant Axonal Neuropathy

Giant Axonal Neuropathy Drugs Uptake and Pipeline Development Activities

The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Giant Axonal Neuropathy market or expected to be launched during the study period. The analysis covers the Giant Axonal Neuropathy market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Giant Axonal Neuropathy drugs based on their sale and market share.

The report also covers the Giant Axonal Neuropathy pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Giant Axonal Neuropathy 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.

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Giant Axonal Neuropathy Therapeutics Analysis

GAN treatment is mainly symptomatic, and the average life expectancy is in the early twenties. 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 physical development and minimize their deterioration as time passes. 

Genetic counseling will be of benefit to patients and their families. Many children with GAN begin with normal intellectual development and are able to attend a regular school program. Children should be scrutinized at least once a year to evaluate their intellectual abilities and to look for the presence of neurological deterioration.

Unfortunately, there is neither a cure nor treatment for the disease. Currently, no drug therapy has been approved for this fatal disorder and ultimately, the patient dies at a very young age.

Emerging and Marketed Giant Axonal Neuropathy Therapies Covered in the Report Include:

At present, Taysha Gene Therapies is involved in the development of a pharmaceutical agent for Giant Axonal Neuropathy.

Taysha Gene Therapies is currently evaluating TSHA-120 (Phase I/II), an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the treatment of GAN. The trial is being conducted by the National Institutes of Health (NIH) in close collaboration with a leading patient advocacy group (Hannah’s Hope) focused on finding treatments and cures for GAN. 

TSHA-120 was granted orphan drug designation from the European Commission in May 2022 and has previously received rare pediatric disease and orphan drug designations from the US FDA for the treatment of GAN.

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Table of Content (TOC)

1. Key Insights

2. Executive Summary 

3. Giant Axonal Neuropathy Competitive Intelligence Analysis

4. Giant Axonal Neuropathy Market Overview at a Glance

5. Giant Axonal Neuropathy Disease Background and Overview

6. Giant Axonal Neuropathy Patient Journey

7. Giant Axonal Neuropathy Patient Population and Epidemiology Trends (In the US, EU5, and Japan)

8. Giant Axonal Neuropathy Treatment Algorithm, Current Treatment, and Medical Practices

9. Giant Axonal Neuropathy Unmet Needs

10. Key Endpoints of Giant Axonal Neuropathy Treatment

11. Giant Axonal Neuropathy Marketed Therapies

12. Giant Axonal Neuropathy Emerging Drugs and Latest Therapeutic Advances

13. Giant Axonal Neuropathy Seven Major Market Analysis

14. Attribute Analysis

15. Giant Axonal Neuropathy Market Outlook (In US, EU5, and Japan)

16. Giant Axonal Neuropathy Companies Active in the Market

17. Giant Axonal Neuropathy Access and Reimbursement Overview

18. KOL Views on the Giant Axonal Neuropathy Market

19. Giant Axonal Neuropathy Market Drivers

20. Giant Axonal Neuropathy Market Barriers

21. Appendix

22. DelveInsight Capabilities

23. Disclaimer

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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About DelveInsight

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