Retinitis pigmentosa (RP) comprises a group of inherited retinal dystrophies characterized
by the degeneration of rod photoreceptors, followed by the degeneration of cone …

Retinal gene supplementation therapy such as the first approved one, voretigene
neparvovec, delivers a functioning copy of the missing gene enabling the protein …

Background CEP290-associated inherited retinal degeneration causes severe early-onset
vision loss due to pathogenic variants in CEP290. EDIT-101 is a clustered regularly …

Many gene therapies are in development for treating people with inherited retinal diseases
(IRD). We hypothesized that potential recipients of gene therapy would have knowledge …

Functional assessments are a fundamental part of the clinical evaluation of patients with
inherited retinal diseases (IRDs). Their importance and impact have become increasingly …

Purpose To validate the use of best‐corrected visual acuity (BCVA), low‐luminance visual
acuity (LLVA), low‐luminance deficit (LLD; the difference between BCVA and LLVA), mean …

Patient-reported outcome measures (PROMs) are tools designed to capture how a patient
feels or functions, without the input or interpretation of anyone else. The earliest PROMs …

Purpose To describe cone structure changes using adaptive optics scanning laser
ophthalmoscopy (AOSLO) in the Rate of Progression of USH2A-related Retinal …

Purpose To investigate the challenges and potential improvement strategies of cost-
effectiveness analyses performed for therapeutics targeting inherited retinal diseases …

Inherited ocular diseases comprise a heterogeneous group of rare and complex diseases,
including inherited retinal diseases (IRDs) and inherited optic neuropathies. Recent success …