LEBER HEREDITARY OPTIC NEUROPATHY
Clinical trials for LEBER HEREDITARY OPTIC NEUROPATHY explained in plain language.
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First human test of gene therapy for blinding eye disease
Disease control CompletedThis early-stage trial tested a new gene therapy called GS010 for Leber Hereditary Optic Neuropathy (LHON), a rare genetic condition that causes vision loss. The main goal was to check if the treatment was safe and tolerable for patients. Researchers gave the therapy to 19 adults…
Matched conditions: LEBER HEREDITARY OPTIC NEUROPATHY
Phase: PHASE1, PHASE2 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Mar 30, 2026 14:34 UTC
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Gene therapy for blindness tracked for 5 years
Disease control CompletedThis study followed 62 people for five years after they received an experimental gene therapy for a rare genetic condition that causes vision loss (Leber Hereditary Optic Neuropathy). The main goal was to check the long-term safety of the treatment and see if any vision improveme…
Matched conditions: LEBER HEREDITARY OPTIC NEUROPATHY
Phase: PHASE3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC
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Rare eye disease study maps patient journey and financial burden
Knowledge-focused CompletedThis study aimed to better understand the progression of Leber Hereditary Optic Neuropathy (LHON), a rare genetic condition that causes sudden vision loss. Researchers collected past medical records and surveyed 44 patients to track how their vision changed over time and how the …
Matched conditions: LEBER HEREDITARY OPTIC NEUROPATHY
Sponsor: GenSight Biologics • Aim: Knowledge-focused
Last updated Mar 18, 2026 14:42 UTC