LEBER HEREDITARY OPTIC NEUROPATHY
Clinical trials for LEBER HEREDITARY OPTIC NEUROPATHY explained in plain language.
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Gene therapy shows promise for rare eye disease that causes blindness
Disease control CompletedThis Phase 3 trial tested a gene therapy called GS010 in 98 people with Leber hereditary optic neuropathy (LHON), a genetic condition that leads to rapid vision loss. Participants received injections of the therapy or a placebo into both eyes. The study measured changes in vision…
Matched conditions: LEBER HEREDITARY OPTIC NEUROPATHY
Phase: PHASE3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Gene therapy shows promise in Long-Term study for rare blindness
Disease control CompletedThis study followed 62 people with Leber Hereditary Optic Neuropathy (LHON), a rare inherited eye disease that causes vision loss, for up to 5 years after they received a single gene therapy treatment called GS010. The goal was to see if the treatment remained safe and if any vis…
Matched conditions: LEBER HEREDITARY OPTIC NEUROPATHY
Phase: PHASE3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Gene therapy for inherited blindness passes early safety check
Disease control CompletedThis study tested a gene therapy called GS010 in 19 adults with Leber Hereditary Optic Neuropathy (LHON), a rare inherited eye disease that causes rapid vision loss. The main goal was to see if the treatment is safe and tolerable at different doses. The therapy uses a harmless vi…
Matched conditions: LEBER HEREDITARY OPTIC NEUROPATHY
Phase: PHASE1, PHASE2 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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New study tracks daily life impact of rare eye disease
Knowledge-focused CompletedThis study looked at 44 people with a rare inherited eye condition called Leber Hereditary Optic Neuropathy (LHON) that causes vision loss. Researchers reviewed medical records and asked participants about their vision, health, and quality of life. The goal was to better understa…
Matched conditions: LEBER HEREDITARY OPTIC NEUROPATHY
Sponsor: GenSight Biologics • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:09 UTC