This article is more than 5 years old. Information may no longer be current.

LHON patients see ‘dramatic, irreversible’ vision decline without treatment

Add topic to email alerts

Receive an email when new articles are posted on

Please provide your email address to receive an email when new articles are posted on .

Subscribe

Added to email alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

Back to Healio

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Back to Healio

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 just how remarkable the visual trends in REVERSE and RESCUE are,” Bernard Gilly, co-founder and CEO of GenSight, said in the release. “The findings will support our effort to provide regulatory authorities with the most robust insights about the natural history of the disease, so that we can convincingly demonstrate the therapeutic benefit delivered by Lumevoq.”