A natural history study shows patients who have Leber’s hereditary optic neuropathy with the ND4 mutation have a dramatic and irreversible decline in visual acuity, a press release from GenSight Biologics said.
The retrospective, cross-sectional REALITY observational study highlights the poor prognosis LHON patients have without treatment, the release said.
In contrast, subjects in the phase 3 REVERSE and RESCUE trials, which treat LHON patients with GenSight’s adeno-associated virus vector gene therapy Lumevoq, have shown visual acuity improvement.
“These findings highlight
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