The report covers emerging Friedreich’s Ataxia drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Friedreich’s Ataxia 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.
Friedreich’s Ataxia: An Overview
Friedreich ataxia (also called FA) is a rare inherited disease that causes progressive nervous system damage and movement problems. It usually begins in childhood and leads to impaired muscle coordination (ataxia) that worsens over time.
It is caused due to a defect (mutation) in a gene labeled FXN, which carries the genetic code for a protein called frataxin. Therefore, individuals who inherit two defective copies of the gene, one from each parent, will develop the disease. FA is characterized by two subtypes, LOFA and VLOFA. For LOFA, the age of onset is between the ages of 26 and 39 years, and for VLOFA, the age of onset is after the age of 40 years.
The symptoms typically begin between the ages of 5 and 15 years. Usually, the first neurological symptom is difficulty walking and poor balance. Another early sign of the disease is slowness and slurring of speech (dysarthria). With time speech becomes hesitant and jerky (often referred to as “scanning of speech”). The difficulty coordinating movement (ataxia) can affect all of the muscles. It gradually worsens and slowly spreads to the arms and the trunk (torso).
Diagnosis of Friedreich’s ataxia requires a careful clinical examination, that includes a thorough physical exam, looking for balance difficulty, loss of joint sensation (proprioception), absence of reflexes, and signs of neurological problems. Moreover, genetic testing now provides a conclusive diagnosis.
Friedreich’s Ataxia Market Key Facts
- According to the National Institute of Neurological Disorders and Stroke, Friedreich ataxia is the most common form of hereditary ataxia in the US, affecting about 1 in every 50,000 people.
- As per the statistics by the National Institute of Neurological Disorders and Stroke, although rare, FA is the most common form of hereditary ataxia in the United States, affecting about 1 in every 50,000 people. Adult or late-onset FA is less common, <25% of diagnosed individuals, and can occur anytime during adulthood.
- According to the study by Jörg S et al. (2009), Friedreich ataxia is the most frequent hereditary ataxia, with an estimated prevalence of 3–4 cases per 100,000 individuals.
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 Friedreich’s Ataxia pipeline therapies. It also thoroughly assesses the Friedreich’s Ataxia 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 Friedreich’s Ataxia 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.
Friedreich’s Ataxia Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Friedreich’s Ataxia epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Friedreich’s Ataxia 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 Friedreich’s Ataxia Epidemiology, Segmented as –
- Total Prevalent Cases of Friedreich’s Ataxia [2019–2032]
- Diagnosed and Treatable Population of Friedreich’s Ataxia [2019–2032]
- Prevalence of Friedreich’s Ataxia based on onset types [2019–2032]
Friedreich’s Ataxia Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in Friedreich’s Ataxia market or expected to be launched during the study period. The analysis covers Friedreich’s Ataxia 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 Friedreich’s Ataxia drugs based on their sale and market share.
The report also covers Friedreich’s Ataxia pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Friedreich’s Ataxia 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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Friedreich’s Ataxia Therapeutics Analysis
Several major pharma and biotech giants are actively working in Friedreich’s Ataxia Market. Currently, Retrotope is leading the therapeutics market with its Friedreich’s Ataxia drug candidates in the most advanced stage of clinical development.
On February 28, 2023, Reata Pharmaceuticals, Inc. (Nasdaq: RETA) announced that the U.S. Food and Drug Administration (“FDA”) had approved SKYCLARYS™ (omaveloxolone) for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. With this approval, the FDA granted a rare pediatric disease priority review voucher.
Friedreich’s Ataxia Companies Actively Working in the Therapeutics Market Include
- AavantiBio
- Design Therapeutics
- Exicure
- Lacerta Therapeutics
- Larimar Therapeutics
- LEXEO Therapeutics
- Metro International Biotech
- Minoryx Therapeutics
- PTC Therapeutics
- Reata Pharmaceuticals
- Retrotope
- StrideBio
- Voyager Therapeutics
And Many Others
Emerging and Marketed Friedreich’s Ataxia Therapies Covered in the Report Include:
- CTI-1601: Larimar Therapeutics
- FXN gene therapy: AavantiBio
- Leriglitazone: Minoryx Therapeutics
- MIB-626: Metro International Biotech
- Omaveloxolone: Reata Pharmaceuticals
- RT001: Retrotope
- SynTEFs: Design Therapeutics
- Vatiquinone: PTC Therapeutics
And Many More
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Friedreich’s Ataxia Competitive Intelligence Analysis
4. Friedreich’s Ataxia Market Overview at a Glance
5. Friedreich’s Ataxia Disease Background and Overview
6. Friedreich’s Ataxia Patient Journey
7. Friedreich’s Ataxia Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Friedreich’s Ataxia Treatment Algorithm, Current Treatment, and Medical Practices
9. Friedreich’s Ataxia Unmet Needs
10. Key Endpoints of Friedreich’s Ataxia Treatment
11. Friedreich’s Ataxia Marketed Therapies
12. Friedreich’s Ataxia Emerging Drugs and Latest Therapeutic Advances
13. Friedreich’s Ataxia Seven Major Market Analysis
14. Attribute Analysis
15. Friedreich’s Ataxia Market Outlook (In US, EU5, and Japan)
16. Friedreich’s Ataxia Companies Active in the Market
17. Friedreich’s Ataxia Access and Reimbursement Overview
18. KOL Views on the Friedreich’s Ataxia Market
19. Friedreich’s Ataxia Market Drivers
20. Friedreich’s Ataxia 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
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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