ADOA causes progressive vision loss that typically begins in childhood. It occurs when a gene mutation (or error) causes the body to make less of a protein called OPA1. OPA1 protein is needed for vision. Without enough OPA1 protein, vision signals cannot travel from the eye to the brain. The study drug, STK-002, is designed to help the body make more OPA1 protein. Making more of this protein may help slow or stop the vision loss caused by ADOA. The goals of the Osprey study are to help researchers find out if the study drug is safe, if it causes any side effects, and how the body processes the study drug.
Symptoms of ADOA include:
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