Leber Hereditary Optic Neuropathy
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CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year [Phase 3]
A Phase 3 trial evaluating bilateral intravitreal GS010 for Leber hereditary optic neuropathy due to the ND4 mutation has completed and post…
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CLINICAL_TRIALS trial_result · 2026-03-02Clinical Trial Update: Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients [Phase 1, Phase 2]
A safety evaluation update from a Phase 1/2 gene therapy trial in Leber hereditary optic neuropathy, reporting safety outcomes without effic…
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CLINICAL_TRIALS withdrawal · 2026-03-02Clinical Trial Update: EAP_GS010_single Patient
The expanded-access program for GS010 in Leber hereditary optic neuropathy is no longer available.
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CLINICAL_TRIALS trial_result · 2026-03-02Clinical Trial Update: REALITY LHON Registry
The REALITY LHON Registry clinical trial, conducted by GenSight Biologics for Leber Hereditary Optic Neuropathy, has completed.
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CLINICAL_TRIALS research · 2026-03-02Clinical Trial Update: Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients [Phase 2]
A Phase 2 clinical trial evaluating bilateral intravitreal injections of GS010 at two dose levels for Leber hereditary optic neuropathy is c…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation [Phase 3]
Phase 3 multicenter, randomized, double-masked, sham-controlled clinical trial for Leber hereditary optic neuropathy due to ND4 mutation. Sp…
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus) [Phase 1, Phase 2]
This is a basket clinical study evaluating glycerol tributyrate in patients with MELAS syndrome or Leber's hereditary optic neuropathy, curr…
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CLINICAL_TRIALS research · 2026-01-07Clinical Trial Update: Tissue Sample Study for Mitochondrial Disorders
Columbia University is enrolling by invitation in a tissue-sample study for mitochondrial disorders, including MELAS, Kearns-Sayre syndrome,…
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CLINICAL_TRIALS research · 2025-12-18Clinical Trial Update: Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy [Phase 1]
Phase 1 pilot trial evaluating nicotinamide (vitamin B3) for efficacy in Leber hereditary optic neuropathy; study status is not yet recruiti…
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CLINICAL_TRIALS research · 2025-11-24Clinical Trial Update: New Non-invasive Modalities for Assessing Retinal Structure and Function [NA]
This clinical trial is evaluating new non-invasive methods for assessing retinal structure and function in ischemic optic neuropathy and rel…
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CLINICAL_TRIALS research · 2025-11-18Clinical Trial Update: Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT) [NA]
An ongoing clinical study (MétabOCT) sponsored by Hôpital Necker-Enfants Malades is investigating metabolomic profiles in relation to the re…
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EMA approval · 2025-08-20EMA Authorised: Raxone (idebenone) — Chiesi Farmaceutici S.p.A
The EMA authorised Raxone (idebenone) from Chiesi Farmaceutici S.p.A. for use in Leber hereditary optic neuropathy (hereditary optic atrophy…
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CLINICAL_TRIALS research · 2025-07-20Clinical Trial Update: Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study
A prospective, multi-center observational trial evaluating optic nerve head blood flow using multimodal imaging in neuro-ophthalmic conditio…
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CLINICAL_TRIALS research · 2025-06-05Clinical Trial Update: Global Registry and Natural History Study for Mitochondrial Disorders
LMU Klinikum is conducting a global registry and natural history study to collect data on mitochondrial disorders, and the study is currentl…
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CLINICAL_TRIALS research · 2025-03-20Clinical Trial Update: Stem Cell Ophthalmology Treatment Study II [NA]
The clinical trial update indicates that the Stem Cell Ophthalmology Treatment Study II is currently recruiting for multiple retinal and opt…
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CLINICAL_TRIALS trial_result · 2024-09-20Clinical Trial Update: Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations [Phase 1, Phase 2]
A Phase 1/2 gene therapy trial for Leber hereditary optic neuropathy caused by ND1 mutations has been terminated.
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CLINICAL_TRIALS research · 2024-09-04Clinical Trial Update: Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations [Phase 1, Phase 2]
Clinical trial update for an ND4-targeted gene therapy for Leber Hereditary Optic Neuropathy (LHON); phases 1 and 2, currently active but no…
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CLINICAL_TRIALS trial_result · 2024-08-07Clinical Trial Update: Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy [Phase 1]
Phase 1 safety study of an adeno-associated virus vector gene therapy for Leber hereditary optic neuropathy has completed enrollment. Result…
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CLINICAL_TRIALS trial_result · 2024-01-25Clinical Trial Update: RESCUE and REVERSE Long-term Follow-up [Phase 3]
Long-term follow-up results from GenSight Biologics' Phase 3 RESCUE and REVERSE trials in Leber hereditary optic neuropathy have been posted…
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CLINICAL_TRIALS trial_result · 2023-09-22Clinical Trial Update: Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy [Phase 2, Phase 3]
A Phase 2/Phase 3 trial evaluating Befizal 200 mg for Leber hereditary optic neuropathy has completed.
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CLINICAL_TRIALS trial_result · 2023-04-21Clinical Trial Update: Study to Assess the Efficacy and Safety of Raxone in LHON Patients [Phase 4]
Phase 4 trial update for Raxone in Leber's Hereditary Optic Neuropathy (LHON) has completed with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2022-11-14Clinical Trial Update: Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy [Phase 2, Phase 3]
Clinical trial update for a gene therapy targeting Leber's Hereditary Optic Neuropathy (LHON) at Phase 2/Phase 3; currently recruiting.
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CLINICAL_TRIALS trial_result · 2021-10-12Clinical Trial Update: A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy [Phase 2]
Phase 2 clinical trial evaluating safety, tolerability, and efficacy of elamipretide topical ophthalmic solution for Leber's Hereditary Opti…
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CLINICAL_TRIALS trial_result · 2020-09-17Clinical Trial Update: A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy [Phase 2, Phase 3]
A clinical trial update for rAAV2-ND4 delivered via a single intravitreal injection in Leber hereditary optic neuropathy, currently in Phase…
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CLINICAL_TRIALS trial_result · 2020-01-30Clinical Trial Update: Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
A completed clinical trial study surveying historical visual acuity data in patients with Leber's Hereditary Optic Neuropathy (LHON); result…