Foundation Fighting Blindness to conduct a natural history study of Usher syndrome type 3
The Foundation Fighting Blindness, funded by the Usher III Initiative, is conducting a natural history study on Usher syndrome type 3 (USH3) caused by the N48K mutation of the CLRN1 gene. This study aims to identify potential patients for future clinical trials, understand disease progression, and optimize clinical trial designs for therapies. The 4-year study will involve up to 20 patients as part of the broader Uni-Rare natural history study, which aims to enroll up to 1,500 patients with inherited retinal diseases (IRD) across multiple sites globally.
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The Foundation Fighting Blindness, funded by the Usher III Initiative, is conducting a natural history study on Usher syndrome type 3 (USH3) caused by the N48K mutation of the CLRN1 gene. This study aims to identify potential patients for future clinical trials, understand disease progression, and optimize clinical trial designs for therapies. The 4-year study will involve up to 20 patients as part of the broader Uni-Rare natural history study, which aims to enroll up to 1,500 patients with inherited retinal diseases (IRD) across multiple sites globally.