LHON


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LHON

Leber's hereditary optic neuropathy, see there.
References in periodicals archive ?
We will continue to work closely with Chiesi Group in the coming months to ensure a smooth transition and a seamless supply of Raxone to LHON patients,' said Thomas Meier, PhD, Chief Executive Officer of Santhera.
Topline 48-week data for RESCUE, the second Phase III clinical trial of GS010 in the treatment of LHON, is expected in early 1Q19.
LHON is caused by mitochondrial DNA mutations in subunits of NADH dehydrogenase (complex I), leading to reduced oxidative phosphorylation and energy production in retinal cells.
The VF testing is essential for making a diagnosis and monitoring the visual function of all optic neuropathies, including LHON. Moreover, VF defects can be the only clinical manifestation of LHON, with no fundoscopic changes.
Raxone is an oral medication, authorized at a daily dose of 900 mg (given as 2 tablets three times a day with food), for the treatment of visual impairment in adolescent and adult patients with LHON.
These increases reflect the expansion of Santhera's operations, especially the commercial activities, the ongoing roll-out of Raxone for LHON across Europe and market entry preparations for Raxone for DMD.
* The report provides a snapshot of the global therapeutic landscape of Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)
"As an organization historically present in Eastern European and Baltic countries with a specific focus on niche indications and diseases with high unmet medical need, we are very excited to have an opportunity to team upwith Santhera to offer for the first time a treatment to patients with LHON," said Alain Staub , CEO and Chairman of the Board of Ewopharma.
LHON is a mitochondrial disease that is caused by well characterized mutations in mitochondrial DNA (mtDNA), with a prevalence of carriers of about 1 in 300 [8].
Based on the recent announcement that its pivotal phase III DELOS trial demonstrated that treatment with Raxone/Catena delays respiratory function loss in patients not using steroids, ValuationLAB expects Santhera to prioritize a new drug application (NDA) for DMD in the US and apply for a variation on the LHON label once approved in the EU.
He tested positive for the 11778G>A mitochondrial mutation associated with LHON.