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Several key players, such as ProQR Therapeutics, Atsena Therapeutics, and others, are actively working in the Leber Congenital Amaurosis therapeutics market to improve the treatment scenario. Few of the therapies have been granted Rare Pediatric Disease and Orphan Drug designation by the US FDA and the European Medicines Agency (EMA).

DelveInsight’s “Leber Congenital Amaurosis Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Leber Congenital Amaurosis market size, share, and trends in the seven major market (7MM) (i.e., the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).

The Leber Congenital Amaurosis market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current 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. 

Leber Congenital Amaurosis: An Overview

Leber congenital amaurosis (LCA) is a rare inherited eye disorder that causes severe vision loss at birth. The disease primarily affects the retina and is the most common cause of inherited blindness in childhood. LCA is inherited in an autosomal recessive manner, meaning that both parents must carry a defective gene for the condition to pass it on to children. There is a 25% chance of inheriting the two LCA genes (one from each parent) needed to cause the disorder.

Leber Congenital Amaurosis Market Key Facts

• According to NORD (2021), the prevalence of Leber congenital amaurosis (LCA) has been estimated to be 1–2 per 100,000 births.

• According to Coussa et al. (2017), LCA is the second most common group of inherited retinal dystrophies after retinitis pigmentosa, accounting for about 5% of all retinal dystrophies. 

• Genetic and Rare Diseases Information Center (2021) estimated that the number of people with LCA in the US is between 3,000 and 30,000. 

Leber Congenital Amaurosis Market

The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Leber Congenital Amaurosis market size by analyzing the impact of current and emerging pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.

The report gives complete detail of the Leber Congenital Amaurosis market trend for each marketed 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.

Leber Congenital Amaurosis Epidemiology Assessment 

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

The Report Covers the Leber Congenital Amaurosis Epidemiology Segmented by – 

• Total Diagnosed Prevalent cases of Leber Congenital Amaurosis in the 7MM [2019–2032]

• Total Prevalent cases of Leber Congenital Amaurosis in the 7MM [2019–2032]

• Gene-specific cases of Leber Congenital Amaurosis in the 7MM [2019–2032]

• Treatable cases of Leber Congenital Amaurosis in the 7MM [2019–2032]

Leber Congenital Amaurosis Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Leber Congenital Amaurosis market or expected to get launched during the study period. The analysis covers the Leber Congenital Amaurosis market uptake by drugs, patient uptake by therapies, and sales of each drug. 

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Leber Congenital Amaurosis Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

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Leber Congenital Amaurosis Therapeutics Analysis

Treatment for Leber Congenital Amaurosis is symptomatic and supportive. Genetic counseling is recommended for families of affected children. Gene therapy aims to halt retinal degeneration by replacing the mutated gene with a normal healthy copy; this enables the affected cells to regain some function and produce functioning proteins. Gene therapy is currently available for LCA caused by two mutations in a gene called RPE65, which accounts for about 6% of LCA cases.

The Leading Companies in the Leber Congenital Amaurosis Therapeutics Market Include:

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Table of Content (TOC)

1. Key Insights

2. Executive Summary 

3. Leber Congenital Amaurosis Competitive Intelligence Analysis

4. Leber Congenital Amaurosis Market Overview at a Glance

5. Leber Congenital Amaurosis Disease Background and Overview

6. Leber Congenital Amaurosis Patient Journey

7. Leber Congenital Amaurosis Epidemiology and Patient Population (In the US, EU5, and Japan)

8. Leber Congenital Amaurosis Treatment Algorithm, Current Treatment, and Medical Practices

9. Leber Congenital Amaurosis Unmet Needs

10. Key Endpoints of Leber Congenital Amaurosis Treatment

11. Leber Congenital Amaurosis Marketed Products

12. Leber Congenital Amaurosis Emerging Drugs and Latest Therapeutic Advances

13. Leber Congenital Amaurosis Seven Major Market Analysis

14. Attribute Analysis

15. Leber Congenital Amaurosis Market Outlook (In US, EU5, and Japan)

16. Leber Congenital Amaurosis Access and Reimbursement Overview

17. KOL Views on the Leber Congenital Amaurosis Market

18. Leber Congenital Amaurosis Market Drivers

19. Leber Congenital Amaurosis Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. 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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Leber Congenital Amaurosis Pipeline Insight

“Leber Congenital Amaurosis Pipeline Insights, 2022” report by DelveInsight outlays comprehensive insights into the present clinical development scenario and growth prospects across the Leber Congenital Amaurosis therapeutics market. Some of the key companies in the Leber Congenital Amaurosis market include ProQR Therapeutics, Editas Medicine, Atsena Therapeutics, and others. 

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