Usher syndrome
MONDO:0019501A syndromic diseae characterized by the association of sensorineural deafness (usually congenital) with retinitis pigmentosa and progressive vision loss.
Also known as: USH, Usher's syndrome, ush, deafness-retinitis pigmentosa syndrome, retinitis pigmentosa-deafness syndrome, Graefe-Usher syndrome, Hallgren syndrome, dystrophia retinae pigmentosa-dysostosis syndrome
16 clinical trials for this condition and its sub-types, 14 tagged with Usher syndrome itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Usher syndrome
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Usher syndrome type 2 2 trials · 3 incl. sub-types
3 sub-types
- Usher syndrome type 2A 1 trial
- Usher syndrome type 2C 0 trials
- Usher syndrome type 2D 0 trials
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Usher syndrome type 1 0 trials · 3 incl. sub-types
9 sub-types
- Usher syndrome type 1B 3 trials
- Usher syndrome type 1C 1 trial
- Usher syndrome type 1D 1 trial
- Usher syndrome type 1F 1 trial
- Usher syndrome type 1G 1 trial
- Usher syndrome type 1E 0 trials
- Usher syndrome type 1H 0 trials
- Usher syndrome type 1K 0 trials
- Usher syndrome, type 1D/F 0 trials
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Usher syndrome type 3 0 trials
2 sub-types
- Usher syndrome type 3A 0 trials
- Usher syndrome type 3B 0 trials
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Usher syndrome, type 4 0 trials
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Can an eye injection slow a genetic cause of blindness?
Disease control Not yet recruitingThis trial tests the long-term safety of an experimental medicine called ultevursen, given as an injection into the eye, for people with retinitis pigmentosa caused by mutations in a gene called USH2A. The study enrolls about 81 adults and children who completed a previous 24-mon…
Phase 2 • Sponsor: Laboratoires Thea • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC
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Can an antioxidant pill slow blindness in usher syndrome?
Disease control Not yet recruitingThis phase 3 trial tests whether an experimental drug called NPI-001 can slow the loss of light-sensing cells in the eyes of adults with Usher syndrome, a genetic condition that causes both hearing and vision loss. About 80 participants take either NPI-001 or a placebo twice dail…
Phase 3 • Sponsor: Nacuity Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jul 19, 2026 00:00 UTC
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New drug aims to preserve sight in rare genetic blindness
Disease control Not yet recruitingThis phase 3 trial tests whether NPI-001, taken twice daily for 24 months, can slow the loss of light-sensing cells in the eyes of adults with Usher syndrome, a genetic condition that causes both hearing and vision loss. About 80 participants will receive either the drug or a pla…
Phase 3 • Sponsor: Nacuity Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC