LHON is most prevalently caused by the MT-ND4 gene mutation in mitochondrial DNA, characterized by acute and severe bilateral ...

“The findings on the persistence of visual improvement show the promise of using LUMEVOQ gene therapy to treat patients with LHON due to the MT-ND4 gene variant,” noted Prof. Patrick Yu-Wai ...

The paper, published in the peer-reviewed journal Survey of Ophthalmology, is the first to compare the efficacy of LHON treatments, approved or in development, on visual outcomes in the ND4-LHON ...

Neurophth is conducting a Phase I/II/III, multi-center, two-parts study aimed at evaluating the safety, tolerability, and efficacy of NR082 in LHON patients with ND4 mutations. The Investigational ...

11778G>A MT-ND4-LHON patients treated with lenadogene nolparvovec ocular gene therapy: The RESTORE study. Presented at: American Academy of Neurology annual meeting; April 22-27, 2023; Boston.

The m.11778G>A MT-ND4 mutation (MT-ND4-LHON) is the most common and is seen in approximately 75% of LHON patients in Europe and North America. It also leads to the most severe form of the disease ...

In the case of the Reflect study, researchers selected patients who had contracted LHON due to a mutation in the NADH dehydrogenase 4 (ND4) protein. Patients were split into two arms, with the ...

Neurophth is conducting a phase I/II, single-arm, multi-center study aimed at evaluating the safety and efficacy of NR082 in LHON patients with ND4 mutations in US. The Investigational New Drug (IND) ...

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Five years after the one-time injection, patients with Leber Hereditary Optic Neuropathy (LHON) due to the MT-ND4 gene variant demonstrated sustained bilateral improvement in visual acuity and ...

Figure 1. “A Gradient of Efficacy”: Visual Recovery (CRR from Nadir) Among ND4-LHON Patients (Graphic: Business Wire) ...