RESCUE and REVERSE Long-term Follow-up

计划入组 62 人 · 首次发布于 2018 年 1 月 23 日

Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)

计划入组 9 人 · 首次发布于 2010 年 12 月 28 日

A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation

计划入组 95 人 · 首次发布于 2026 年 2 月 12 日

LHON-naphthoquinone-related small molecules(University of Cardiff)

AAV2 smCBA based ND4 gene therapy (University of Miami)

AAV2 smCBA based G11778A ND4 gene therapy (University of Miami)

Ndc Inc

United States

LION CORPORATION

Japan

BENTA LYON

France

GenSight's Gene Therapy Shows Better Outcomes When Administered During Dynamic Phase of LHON Disease

GenSight Biologics Secures French Regulatory Approvals for GS010/LUMEVOQ Gene Therapy in Rare Blindness Disease

GenSight Biologics Secures FDA Authorization for Expanded Access Treatment of Rare Blinding Disease