DelveInsight's "Leber Congenital Amaurosis Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of the Leber Congenital Amaurosis, historical and forecasted epidemiology as well as the Leber Congenital Amaurosis market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
The Leber Congenital Amaurosis market report provides current treatment practices, emerging drugs, Leber Congenital Amaurosis market share of the individual therapies, and current and forecasted Leber Congenital Amaurosis market Size from 2019 to 2032 segmented by seven major markets. The Report also covers current Leber Congenital Amaurosis treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the Leber Congenital Amaurosis market.
Do you know about Leber Congenital Amaurosis Market Size?
Leber Congenital Amaurosis (LCA) is a rare genetic eye disorder. Affected infants are often blind at birth. Other symptoms may include crossed eyes (strabismus); rapid, involuntary eye movements (nystagmus); unusual sensitivity to light (photophobia); clouding of the lenses of the eyes (cataracts); and/or a cone shape to the front of the eye (keratoconus). LCA is usually inherited as an autosomal recessive genetic condition.
Children born with LCA have light-gathering cells (rods and cones) of the retina that do not function properly. The absence or reduction of the electrical activity of the retina is always observed and is necessary for the diagnosis of LCA. A decrease in visual responsiveness at birth is the first sign of the disease. Often the child will poke, press, and rub the eyes to stimulate the retina to produce light (Franceschetti’s oculo-digital sign). This activity may cause the eyes to become sunken or deep-set (enophthalmos). LCA is a monogenic disease and at least 27 genes are implicated. Changes (mutations) in these genes can account for about 80-90% of diagnosed cases of LCA. The genes responsible for the remaining 10-20% of diagnoses are not known. LCA is usually inherited as an autosomal recessive genetic condition.
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What are the Leber Congenital Amaurosis Emerging Drugs?
Sepofarsen (QR-110): ProQR Therapeutics
Sepofarsen (QR-110) is an RNA therapy that aims to restore vision in Leber Congenital Amaurosis 10 (LCA10) due to the most common p.Cys998X mutation in the CEP290 gene. Sepofarsen aims to restore vision loss in people with Leber Congenital Amaurosis due to a specific mutation in the CEP290 gene. This p.Cys998X mutation, also known as c.2991+1655A>G, is the most common mutation causing LCA10. The p.Cys998X mutation causes a mistake in the CEP290 RNA and therefore a process called splicing is not done properly. The cell can therefore not use the RNA to produce a working CEP290 protein that is essential for vision. Currently, it is in Phase II/III stage of development to treat Leber Congenital Amaurosis.
EDIT-101: Editas Medicine, Inc.
The company’s approach to Leber Congenital Amaurosis 10 (LCA10) is to target a disease-causing mutation in the CEP290 gene that causes degeneration in ocular photoreceptor cells—cells critical for experiencing normal vision. They have developed an experimental CRISPR medicine called EDIT-101 designed to remove the CEP290 mutation. This approach may restore normal protein expression, photoreceptor function, and ultimately, vision.
What is the Leber Congenital Amaurosis Market Outlook?
The Leber Congenital Amaurosis market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Leber Congenital Amaurosis market trends by analyzing the impact of current Leber Congenital Amaurosis therapies on the market, unmet needs, drivers and barriers, and demand for better technology.
This segment gives a thorough detail of Leber Congenital Amaurosis market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Leber Congenital Amaurosis market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
What is the Leber Congenital Amaurosis Market Report Scope?
· The report covers the descriptive overview of Leber Congenital Amaurosis, explaining its causes, signs and symptoms, pathophysiology, diagnosis, and currently available therapies
· Comprehensive insight has been provided into the Leber Congenital Amaurosis epidemiology and treatment in the 7MM
· Additionally, an all-inclusive account of both the current and emerging therapies for Leber Congenital Amaurosis is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
· A detailed review of the Leber Congenital Amaurosis market forecast; historical and forecasted is included in the report, covering drug outreach in the 7MM
· The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Leber Congenital Amaurosis market
What is the Leber Congenital Amaurosis Market Report Highlights?
· In the coming years, the Leber Congenital Amaurosis market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
· The companies and academics are working to assess challenges and seek opportunities that could influence Leber Congenital Amaurosis R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
· Major players are involved in developing therapies for Leber Congenital Amaurosis. The launch of emerging therapies will significantly impact the Leber Congenital Amaurosis market
· A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Leber Congenital Amaurosis
· Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.
Also, read- Leber Congenital Amaurosis Market Companies
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