Inherited retinal diseases: Therapeutics, clinical trials and end points—A review

نویسندگان

چکیده

Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of disorders characterised by photoreceptor degeneration or dysfunction. These typically present with severe vision loss that can be progressive, disease onset ranging from congenital to late adulthood. The advances in genetics, imaging molecular biology, have conspired create the ideal environment for establishing treatments IRDs, first approved gene therapy commencement multiple clinical trials. scope this review is familiarise clinicians scientists current management prospects novel therapies for: (1) macular dystrophies, (2) cone cone-rod (3) dysfunction syndromes, (4) Leber amaurosis, (5) rod-cone (6) rod syndromes (7) chorioretinal dystrophies. We also briefly summarise investigated end points ongoing

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ژورنال

عنوان ژورنال: Clinical and Experimental Ophthalmology

سال: 2021

ISSN: ['1442-6404', '1442-9071']

DOI: https://doi.org/10.1111/ceo.13917