X-LINKED RETINITIS PIGMENTOSA
Clinical trials for X-LINKED RETINITIS PIGMENTOSA explained in plain language.
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One-Shot gene injection aims to halt blindness in rare eye disease
Disease control CompletedThis study tested whether a single injection of an experimental gene therapy could slow or stop vision loss in people with X-linked retinitis pigmentosa, a rare inherited eye disease. The therapy, called AAV5-hRKp.RPGR, delivers a working copy of a faulty gene directly into the e…
Matched conditions: X-LINKED RETINITIS PIGMENTOSA
Phase: PHASE3 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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Five-Year watch on Vision-Saving gene therapy
Disease control CompletedThis study followed 42 adults and children for up to 5 years after they received an experimental gene therapy for a genetic eye disease called X-linked retinitis pigmentosa. The main goal was to check for any long-term safety issues from the treatment. Researchers also tracked wh…
Matched conditions: X-LINKED RETINITIS PIGMENTOSA
Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Mar 24, 2026 12:02 UTC