Leber congenital amaurosis
Tracked across 1 events · 7 articles · First seen Apr 22, 2026 · Last active Apr 22, 2026
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Impact: Leber congenital amaurosis is the rare inherited condition that Saffie Sandford suffers from, which the Voretigene neparvovec gene therapy aims to treat.
Apr 22, 2026 · 7 articles
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