XLRP3 affects retinal structure and function symmetrically, supporting the use of the fellow eye as an internal control in interventional trials. VA and kinetic visual fields (III4e) seem promising functional outcome measures to assess disease progression. KMC analysis predicted the most severe decline in vision between the third and fourth decade of life.
In future we can expect gene therapy to continue to become more clinically relevant. More than ever, it is now essential to generate precise characterizations of the natural disease progression of IRDs through observational or retrospective studies in order to guarantee a most effective study design.
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