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Las Vegas (Nevada), United States //— The Leber Congenital Amaurosis Market is set to register immense growth in the coming years owing to the increasing prevalent population of Leber congenital amaurosis patients in the 7MM and the launch of novel therapies in the market. Various pharmaceutical companies have shifted their focus towards the development of new treatment therapies for this rare indication.
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 markets (7MM) (i.e., the United States, EU5 (Germany, Spain, Italy, France, and the 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.
Babies born with LCA have a very reduced vision that can often be detected by parents in the first few months of life. They may not respond to visual cues and have roving eye movements, called nystagmus. Many children with LCA habitually press on their eyes with their fists or fingers, which causes their eyes to look sunken or
deep-set.
Some of the major pharma and biotech companies such as ProQR Therapeutics, Atsena Therapeutics, and others are involved in developing new therapies to treat LCA.
Leber Congenital Amaurosis Market Key Facts
– According to National Organization for Rare Disorders (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. With an estimated prevalence ranging from 1 per 33,000 to 1 per 81,000, LCA was reported to account for about 20% of legal blindness in children.
– 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.
– As per American Association for Pediatric Ophthalmology & Strabismus (2022), LCA is rare (2–3 cases per 100,000 births); about 10–18% of all cases of congenital blindness or severely reduced vision in children are caused by LCA.
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 pools, future trends, 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]
Learn More About the Evolving Epidemiology Trends @ Leber Congenital Amaurosis Epidemiology Analysis
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 analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Explore More About the Pipeline Development Activities @ Leber Congenital Amaurosis Therapeutics Pipeline Assessment
Leber Congenital Amaurosis Therapeutics Analysis
Treatment for LCA 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.
Several major pharma and biotech giants are developing the therapies for Leber Congenital Amaurosis. Currently, ProQR Therapeutics is leading the therapeutics market with its Leber Congenital Amaurosis drug candidates in the most advanced stage of clinical development.
Some of the Leading Companies in the Leber Congenital Amaurosis Therapeutics Market Include:
– ProQR Therapeutics
– Editas Medicine
– Atsena Therapeutics Inc.
– Applied Genetic Technologies Corporation
– IVERIC bio
– MeiraGTx
– Spark Therapeutics
And many others
Leber Congenital Amaurosis Therapies Covered in the Report Include:
– Sepofarsen (QR-110): ProQR Therapeutics
– EDIT-101: Editas Medicine, Inc.
– ATSN-101: Atsena Therapeutics
– Luxturna: Spark Therapeutics
And many more
Get a more detailed analysis @ Leber Congenital Amaurosis Emerging Therapies and Key Companies
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. 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.
Learn how the Leber Congenital Amaurosis market will evolve by 2032 @ Leber Congenital Amaurosis Market Outlook 2032
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Shruti Thakur
Manager (Marketing and Branding)
Email: info@delveinsight.com
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