Inherited retinal diseases: therapeutics, clinical trials and end points—a review

M Georgiou, K Fujinami… - Clinical & Experimental …, 2021 - Wiley Online Library
Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of
disorders characterised by photoreceptor degeneration or dysfunction. These disorders
typically present with severe vision loss that can be progressive, with disease onset ranging
from congenital to late adulthood. The advances in genetics, retinal imaging and molecular
biology, have conspired to create the ideal environment for establishing treatments for IRDs,
with the first approved gene therapy and the commencement of multiple clinical trials. The …

[PDF][PDF] Inherited retinal diseases: Therapeutics, clinical trials and end-points–A review

K Fujinami, M Michaelides - researchgate.net
Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of
disorders characterised by photoreceptor degeneration or dysfunction. These disorders
typically present with severe vision loss which can be progressive, with disease onset
ranging from congenital to late adulthood. The advances in genetics, retinal imaging and
molecular biology, have conspired to create the ideal environment for establishing
treatments for IRD, with the first approved gene therapy and the commencement of multiple …
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