Mitochondrial nt3243 A>G Mutation in Taiwan

计划入组 40 人 · 首次发布于 2014 年 4 月 15 日

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

计划入组 39 人 · 首次发布于 2016 年 1 月 12 日

Maternal Inheritance of a Pathogenic MT-ND1 Mutation Causes Mitochondrial Dysfunction and Spermatogenic Failure in Men

计划入组 1,200 人 · 首次发布于 2026 年 7 月 9 日

Mitochondrial protonophores (Gencia)

Controlled-release mitochondrial protonophore

Rare mitochondrial diseases(MitoCareX)

Reneo Pharmaceuticals

United States

Cogent Biosciences

United States

Nuvation Bio

United States

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

LUMEVOQ Gene Therapy Shows Sustained Vision Improvement in LHON Patients After 5 Years