LEBER HEREDITARY OPTIC NEUROPATHY (OPTIC, ATROPHY, HEREDITARY, LEBER)

Clinical trials for LEBER HEREDITARY OPTIC NEUROPATHY (OPTIC, ATROPHY, HEREDITARY, LEBER) explained in plain language.

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Experimental gene therapy offered to single patient with rare blinding condition

Disease control NO_LONGER_AVAILABLE

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 …

Matched conditions: LEBER HEREDITARY OPTIC NEUROPATHY (OPTIC, ATROPHY, HEREDITARY, LEBER)

Sponsor: GenSight Biologics • Aim: Disease control

Last updated Mar 25, 2026 14:08 UTC

LEBER HEREDITARY OPTIC NEUROPATHY (OPTIC, ATROPHY, HEREDITARY, LEBER)

Get alerted when new LEBER HEREDITARY OPTIC NEUROPATHY (OPTIC, ATROPHY, HEREDITARY, LEBER) trials appear

Experimental gene therapy offered to single patient with rare blinding condition