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Clinical Trials
66
4 active
Approvals
43
Total approvals
Agencies
2
Regulatory bodies
Founded
1994
Active, not recruiting
3.0%
Completed
50
75.8%
Not yet recruiting
Recruiting
6
9.1%
Terminated
5
7.6%
Withdrawn
1
1.5%
CANADA
Health Canada
4
CIMA_AEMPS
Spanish Agency for Medicines and Medical Devices
39
- The Usher Syndrome market is poised for growth with emerging therapies addressing the root cause of the disease, offering alternatives to supportive care. - Ultevursen, an investigational RNA therapy, targets the USH2A gene mutation and is currently in Phase II/III trials, showing promise in restoring functional usherin protein. - Nacuity Pharmaceuticals' NPI-001, a free radical scavenger, is in Phase I/II trials, aiming to reduce oxidative stress and preserve photoreceptor cells in Usher Syndrome patients. - The US has the highest prevalent cases of Usher Syndrome, with approximately 21,000 cases in 2023, highlighting the significant unmet need for effective treatments.
