Experimental gene therapy offered to single patient with rare blinding condition
NCT ID NCT03672968
Summary
This was an expanded access program that provided an experimental gene therapy called GS010 to one patient with Leber hereditary optic neuropathy, a rare genetic condition that causes vision loss. The therapy involved injections into both eyes and aimed to control the disease by addressing its genetic cause. The patient had a specific genetic mutation and was not eligible for other ongoing clinical trials.
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