Usher Syndrome Treatment Market Size in 7MM is expected to grow at a decent CAGR by 2034, estimates DelveInsight

DelveInsight's " Usher Syndrome Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of Usher Syndrome, historical and forecasted epidemiology as well as the Usher Syndrome market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

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Key Takeaways from the Usher Syndrome Market Report
• Among the 7MM, the US accounted for the highest prevalent cases of Usher Syndrome in 2023, with around 21,000 cases; these cases are expected to increase during the forecast period.
• Amongst EU4 and the UK, the total prevalent cases of Usher Syndrome were highest in Germany, while the lowest number of cases were in Spain in 2023.
• According to the estimates, in the US, it is observed that type 2 of Usher Syndrome was most prevalent type, accounting for over 60% of total cases in 2023.
• The leading Usher Syndrome Companies such as Laboratoires Théa, Nacuity Pharmaceuticals, AAVantgarde Bio, Odylia Therapeutics, Eli Lilly and Company, Atsena Therapeutics, Clearside Biomedical, and others.
• Promising Usher Syndrome Therapies such as Ultevursen, NPI-001, AAV-USH1C, and others.

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Usher Syndrome Epidemiology Segmentation in the 7MM
• Total Usher Syndrome Prevalent Cases
• Usher Syndrome Diagnosed Prevalent Cases
• Usher Syndrome Type-specific Cases

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Emerging Usher Syndrome Drugs Profile

• NPI-001: Nacuity Pharmaceuticals
NPI-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA) being developed to treat a broad set of diseases and conditions associated with oxidative stress and reduced glutathione levels. In preclinical animal studies, NPI-001 preserved photoreceptor cells and functionality. A Phase I clinical trial of Nacuity's GMP-grade NPI-001 solution in healthy volunteers was completed with no serious adverse events. Currently, the company is investigating iptacopan in the Phase I/II trial (NCT04355689) for the treatment of patients with RP (retinitis pigmentosa) associated with Usher Syndrome.

• Ultevursen: Laboratoires Théa
Ultevursen (formerly QR-421a) is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher Syndrome type 2a and non-syndromic USH due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional usherin protein by using an exon-skipping approach with the aim of stopping or reversing vision loss in patients. Ultevursen is intended to be administered through intravitreal injections in the eye and has been granted Orphan Drug Designation in the US and the European Union and received Fast Track and Rare Pediatric Disease Designations from the FDA. Currently, Ultevursen is being investigated in a Phase II/III trial, SIRIUS, for treating Usher Syndrome Type 2.

Usher Syndrome Market Outlook
LUXTURNA (voretigene neparvovec) is the only approved therapy for Usher Syndrome and is only authorized for the treatment of a small subpopulation of patients that have the RPE65 mutation. There is currently no standard treatment for patients without the RPE65 mutation; therefore, most patients are limited to the best supportive care, including reliance on vitamin supplements, protection from sunlight, and visual aids. Only few key players are active in the pipeline such as Nacuity Pharmaceuticals and Laboratoires Théa for the treatment of Usher Syndrome.

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Usher Syndrome Treatment Market
Treatment involves managing hearing, vision, and balance problems. Some researchers have shown that taking a specific daily dose of vitamin A may slow the progression of retinal degeneration in some people with typical RP and Usher Syndrome type 2. Typically, treatment for hearing management includes hearing aids, assistive listening devices, cochlear implants, or other communication methods. Although some ophthalmologists believe that a high dose of vitamin A palmitate may slow, but not halt the progression of retinitis pigmentosa. Other treatments such as gene therapy, drug based therapy, artificial organ replacement are currently leading the market. Ongoing research on the Usher proteins function in auditory and visual cells will be important in determining the optimal target for gene replacement therapy.

Usher Syndrome Drugs Market
Free radical scavengers play a role in the treatment of Usher Syndrome by helping to reduce oxidative stress, which is implicated in the progression of the disease. Usher Syndrome is a genetic condition characterized by hearing loss and vision impairment, including retinitis pigmentosa. Research has shown that free radical scavengers, such as minocycline, nanoceria, and edaravone, possess antioxidant properties that can help reduce reactive oxygen species (ROS) levels and increase the expression of antioxidant-associated proteins in the retina. This can potentially contribute to the preservation of retinal function in conditions like retinitis pigmentosa, which is a major hallmark of Usher Syndrome.

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Scope of the Usher Syndrome Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Usher Syndrome Companies- Laboratoires Théa, Nacuity Pharmaceuticals, AAVantgarde Bio, Odylia Therapeutics, Eli Lilly and Company, Atsena Therapeutics, Clearside Biomedical, and others.
• Usher Syndrome Therapies- Ultevursen, NPI-001, AAV-USH1C, and others.
• Usher Syndrome Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Usher Syndrome Unmet Needs, KOL's views, Analyst's views, Usher Syndrome Market Access and Reimbursement

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Table of Content
1. KEY INSIGHTS
2. REPORT INTRODUCTION
3. EXECUTIVE SUMMARY OF USHER SYNDROME (USH)
4. USHER SYNDROME MARKET OVERVIEW AT A GLANCE
5. KEY EVENTS
6. EPIDEMIOLOGY AND MARKET FORECAST METHODOLOGY
7. USHER SYNDROME: DISEASE BACKGROUND AND OVERVIEW
8. EPIDEMIOLOGY AND PATIENT POPULATION
9. PATIENT JOURNEY
10. MARKETED DRUGS
11. EMERGING DRUGS
12. USHER SYNDROME: MARKET ANALYSIS
13. UNMET NEEDS
14. SWOT ANALYSIS
15. MARKET ACCESS AND REIMBURSEMENT
16. APPENDIX
17. DELVEINSIGHT CAPABILITIES
18. DISCLAIMER
19. ABOUT DELVEINSIGHT

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