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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A GeneAbnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deaf-Blind Disorders, Deafness, Ear Diseases, Eye Abnormalities, Eye Diseases, Hereditary, Hearing Loss, Sensorineural, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, Usher syndrome, Usher syndrome type 2, blindness (disorder), eye disorder, hearing disorder, hearing loss disorder, hereditary disease, inherited retinal dystrophy, nervous system disorder, otorhinolaryngologic disease, retinal degeneration, retinal disorder, retinitis pigmentosa, vision disorderActive, not recruitingPhase 22026-07-09NCT06627179
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Abnormalities, Eye Diseases, Hereditary, Leber congenital amaurosis, Leber congenital amaurosis 10, Leber hereditary optic neuropathy, Meckel syndrome, type 4, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, blindness (disorder), cranial nerve neuropathy, eye disorder, hereditary disease, hereditary optic atrophy, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, optic atrophy, optic nerve disorder, retinal disorder, vision disorderActive, not recruitingPhase 2 / Phase 32022-03-17NCT03913143