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Leber Congenital Amaurosis Market is Expected to Show a Healthy Growth Rate During the Study Period (2019-2032)| Key Companies - ProQR Therapeutics, Atsena Therapeutics, Editas Medicine
Leber Congenital Amaurosis Market
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Leber Congenital Amaurosis Overview
Leber Congenital Amaurosis 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.
Leber Congenital Amaurosis 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 Leber Congenital Amaurosis genes (one from each parent) needed to cause the disorder.
Babies born with Leber Congenital Amaurosis 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 Leber Congenital Amaurosis habitually press on their eyes with their fists or fingers, which causes their eyes to look sunken or deep-set.
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Leber Congenital Amaurosis Market Report Key Highlights
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.
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.
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.
Leber Congenital Amaurosis Treatment Market
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.
Treatment with Luxturna is not recommended for patients younger than 12 months of age because the retina is still growing, which may affect how Luxturna works
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The Report Covers the Leber Congenital Amaurosis Epidemiology Segmented by:
Leber Congenital Amaurosis Incident Cases
Leber Congenital Amaurosis treatment cases
Leber Congenital Amaurosis prevalent cases
Leber Congenital Amaurosis diagnosed cases
Leber Congenital Amaurosis Therapy Assessment
ProQR Therapeutics: Sepofarsin
Atsena Therapeutics: ATSN-101
Editas Medicine: EDIT-101
And many others.
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Table of Contents
Key Insights
Leber Congenital Amaurosis Introduction
Executive Summary of Leber Congenital Amaurosis
Disease Background and Overview
Epidemiology and patient population
Leber Congenital Amaurosis Emerging Therapies
Leber Congenital Amaurosis Market Outlook
Market Access and Reimbursement of Therapies
Market Drivers
Market Barriers
Appendix
Report Methodology
DelveInsight Capabilities
Disclaimer
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DelveInsight Business Research LLP
Adya Kaul
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https://www.delveinsight.com
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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.
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