30 closest matches · ranked by relevance
Leber's Hereditary Optic Neuropathy
Primary: Double Masked Period: Incidence of Ocular TEAEs — 3; 8; 1; 3 eyes
Leber's Hereditary Optic Neuropathy (LHON)
Primary: In Eyes With VA Assessment Made ≤1 Year After Onset of Symptoms: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA (Measured by Change in ETDRS Letters)…
Leber Hereditary Optic Neuropathy
Primary: The Best Corrected Visual Acuity(BCVA) — 1.40; 1.69; 1.36; 1.58 logMAR
Leber's Hereditary Optic Neuropathy
Primary: Number of Treatment Related Adverse Events — 1; 1; 1; 5 Participants
Optic, Atrophy, Hereditary, Leber
Primary: Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48 — 0.380; 0.392 LogMAR — p=0.8890
Optic, Atrophy, Hereditary, Leber
Primary: Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48 — -0.219; -0.211 LogMAR — p=0.8783
Leber Hereditary Optic Neuropathy
Primary: Change From Baseline of the Best Corrected Visual Acuity (BCVA) Reported With Log of the Minimal Angle of Resolution (LogMAR) at 1.5 Years Post-treatment, in the Second…
Leber Congenital Amaurosis
Primary: Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event — 0; 0; 1; 0 Participants
MELAS · LHON · Leigh Syndrome
Primary: SAD: Change From Baseline in ECG Results by Timepoint: Corrected QT Interval According to Fridericia's Formula (QTcF) — 386.2; 395.0; 385.8; 399.3 msec
Non-arteritic Ischemic Optic Neuropathy · Optic Nerve Injuries
Primary: Mean Increase From Baseline in ETDRS Letters Read at 26 Weeks and Off-drug Follow-up Visit — 15; 7; 9; 6 EDTRS Letters
Non-arteritic Ischemic Optic Neuropathy
Primary: Change From Baseline in Visual Acuity at 9 Month — -0.0574; 0.0535 LogMAR
Leber's Congenital Amaurosis
Primary: Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes — 6; 5 Participants
Leber's Hereditary Optic Neuropathy (LHON)
Primary: Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline — 60 eyes — p=0.0021
Nonarteritic Anterior Ischemic Optic Neuropathy
Primary: Change in Visual Acuity — 4; 3 lines change in Snellen chart
Leber Congenital Amaurosis
Primary: Number of Participants Experiencing Ocular or Non-ocular Adverse Events — 6; 6 participants
Glaucoma
Primary: Lamina Cribrosa (Boundaries of the Retinal Layers) Measurements — 264.3; 273.3; 276.2 micro-ohms
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Age-related Macular Degeneration
Primary: Change in Best Corrected Visual Acuity — 6.0; 4.6 Change in letters (BCVA)
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Hereditary Sensory and Autonomic Neuropathy Type I
Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale
Retinitis Pigmentosa · X-linked Genetic Diseases
Primary: Rate of LOSS of 31 Hertz Cone Electroretinographic Function — 0.028; 0.022 log microvolts/year — p=0.30
Leber Hereditary Optic Neuropathy
Primary: Ocular Adverse Events (AEs) — 5; 1 eyes
Anterior Ischemic Optic Neuropathy · Ischemic Optic Neuropathy
Primary: Crude Incidence Rate of Non-arteritic Anterior Ischemic Optic Neuropathy (NAION) Per 1000 Person-years by Age — 0.01; 0.01; 0.03; 0.12 incidence rate per 1000 person-years
Retinal Dystrophies · Healthy
Primary: Change in the Planimetric Area of Goldmann Visual Field With the Use of Head-mounted Display Technology Compared to Baseline (Measured in Degrees Squared) — 563.08…
Neurofibromatosis 1
Primary: Mitochondrial Respiration Efficiency (as Measured by OCR). — 67.7167; 53.7000; 73.3358; 68.5789 picomole/minute — p=<0.01
Growth Hormone Deficiency · Septo-Optic Dysplasia · Hypopituitarism
Primary: Change in Anthropometric Measures Over Time — 1.97; -0.61; 1.39; 1.22 Z-score
Age-related Macular Degeneration
Primary: Change in Best Corrected Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Visual Scale Testing — 4.5 Change in letters (BCVA)
Albinism
Primary: Improved Vision — 0.67; 0.55; 0.53 logMAR
Retinitis Pigmentosa
Primary: ETDRS Visual Acuity (High Contrast) — -0.02; 0.11 log unit
Optic Neuritis
Primary: Retinal Ganglion Cell Inner Plexiform Layer Thickness — 36.56; 34.65; 31; 29.42 micron