Usher syndrome Market

Emerging therapies for Usher syndrome, including NPI-001, Ultevursen, and others, are anticipated to drive growth in the Usher syndrome market over the coming years.

DelveInsight has released a new report titled “Usher Syndrome – Market Insights, Epidemiology, and Market Forecast–2034”, which provides a comprehensive analysis of the disorder, covering historical and projected epidemiology, along with market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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Some of the key facts of the Usher syndrome Market Report:

• According to DelveInsight, the overall Usher Syndrome market across the 7MM was valued at approximately USD 77 million in 2023 and is expected to grow at a notable CAGR throughout the forecast period (2020–2034).

• The United States accounted for the largest patient pool in 2023, with nearly 21,000 diagnosed cases—a figure projected to increase in the coming years. While leading companies such as Eli Lilly, Atsena Therapeutics, and Nacuity Pharmaceuticals are advancing innovative treatment approaches, the current market continues to rely heavily on off-label therapies that fail to address the root cause. Supportive measures, including vitamin supplementation, sun protection, and visual aids, remain essential components of patient care.

• At present, Luxturna is the only approved therapy for retinal degeneration, a hallmark feature of Usher Syndrome. In January 2020, the FDA granted Rare Pediatric Disease Designation to QR-421a for treating exon 13 mutations of the USH2A gene. More recently, in December 2024, AAVB-081, a dual AAV8-based gene therapy targeting MYO7A mutations linked to Usher Syndrome Type 1B (USH1B), received Orphan Drug Designation, offering incentives such as tax credits, fee exemptions, and up to seven years of market exclusivity upon approval.

• Looking ahead, several promising therapies—including NPI-001, Ultevursen, and others—are expected to shape the future treatment landscape of Usher Syndrome.

What is Usher syndrome?

Usher Syndrome is the most common genetic cause of deaf-blindness, responsible for nearly 50% of cases in individuals younger than 65. This autosomal recessive disorder impacts hearing, vision—primarily due to retinitis pigmentosa—and balance. The condition manifests with diverse onset patterns and severities, frequently involving balance impairments and bilateral vestibular areflexia. Clinically, Usher Syndrome is categorized into three main types—Type 1, Type 2, and Type 3—each associated with distinct genetic mutations and further subdivided into additional subtypes.

Usher syndrome Market Outlook

At present, LUXTURNA (voretigene neparvovec) is the only approved therapy related to Usher Syndrome, targeting patients with the RPE65 mutation, which leaves the majority without a definitive treatment option. Most individuals depend on supportive measures such as vitamin supplementation, sun protection, and visual aids.

Only a limited number of companies, including Nacuity Pharmaceuticals and Laboratoires Théa, are pursuing therapeutic development. While high doses of vitamin A palmitate and fish oil are often recommended, clinical studies indicate minimal or no proven benefit, and vitamin E may even hinder vitamin A absorption.

Consequently, there remains no established cure or effective therapy capable of stopping or reversing disease progression in most patients with Usher Syndrome.

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Usher syndrome Emerging Drugs

• NPI-001: Nacuity Pharmaceuticals

• Ultevursen: Laboratoires Théa

Scope of the Usher syndrome Market Report

• Study Period: 2020-2034

• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

• Key Usher syndrome Companies: Atsena Therapeutics, ProQR Therapeutics, Editas Medicine, Nacuity Pharmaceuticals, and others

• Key Usher syndrome Therapies: NPI-001, Ultevursen, and others

• Usher syndrome Therapeutic Assessment: Usher syndrome current marketed and Usher syndrome emerging therapies

• Usher syndrome Market Dynamics: Usher syndrome market drivers and Usher syndrome market barriers

• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies

• Usher syndrome Unmet Needs, KOL’s views, Analyst’s views, Usher syndrome Market Access and Reimbursement

To know what’s more in our Usher syndrome report, visit https://www.delveinsight.com/report-store/usher-syndrome-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Key benefits of the Usher syndrome Market Report:

1. Usher syndrome market report covers a descriptive overview and comprehensive insight of the Usher syndrome Epidemiology and Usher syndrome market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).

2. The Usher syndrome market report provides insights into the current and emerging therapies.

3. The Usher syndrome market report provides a global historical and forecasted market covering drug outreach in 7MM.

4. The Usher syndrome market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Usher syndrome market.

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Table of Contents

1. Report Introduction

2. Executive Summary

3. SWOT analysis

4. Usher syndrome Patient Share (%) Overview at a Glance

5. Usher syndrome Market Overview at a Glance

6. Usher syndrome Disease Background and Overview

7. Usher syndrome Epidemiology and Patient Population

8. Country-Specific Patient Population of Usher syndrome

9. Usher syndrome Current Treatment and Medical Practices

10. Unmet Needs

11. Usher syndrome Emerging Therapies

12. Usher syndrome Market Outlook

13. Country-Wise Usher syndrome Market Analysis (2020–2034)

14. Market Access and Reimbursement of Therapies

15. Market drivers

16. Market barriers

17. Appendix

18. Usher syndrome Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

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Related Reports:

Usher syndrome Pipeline Insights, DelveInsight

“Usher syndrome Pipeline Insight, 2024” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Usher syndrome market. A detailed picture of the Usher syndrome pipeline landscape is provided, which includes the disease overview and Usher syndrome treatment guidelines.

About DelveInsight

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.

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