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LHONLeber's Hereditary Optic Neuropathy
LHONLeber Hereditary Optic Neuroretinopathy
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As per the agreement, Chiesi Group has in-licensed Raxone for the treatment of Leber's hereditary optic neuropathy (LHON) and the initial payment of EUR 44 million of the total consideration of up to EUR 93 million became due.
In addition, large improvements were also noted in other domains relevant to LHON patients: role difficulties, general vision, and overall mental health.
Also, BridgeBio announced that it has launched a subsidiary company Fortify Therapeutics to further develop this chemistry for local treatment of Leber's Hereditary Optic Neuropathy (LHON), with an initial financial commitment of USD20m.
Patients with LHON have typical and consistent clinical findings that include loss of visual acuity, dyschromatopsia, decreased contrast sensitivity, and visual field (VF) defects [6].
Following the European Commission's marketing authorization for Raxone, the first approved treatment in LHON and the first approved treatment for a mitochondrial disease, the Ministry of Health Israel granted Raxone a marketing authorization for the treatment of visual impairment in adolescent and adult patients with LHON.
In the first six months of 2016, Raxone generated net sales of CHF 7.2 million (1H 2015: CHF 1.5 million; 2H 2015: CHF 2.8 million), mainly driven by increased Raxone sales to LHON patients in Germany and France.
The report provides comprehensive information on the therapeutics under development for Leber's Hereditary Optic Neuropathy (LHON) (Leber optic atrophy), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
LHON is a uncommon, hereditary, mitochondrial disease, which leads to fast, profound and usually permanent blindness in otherwise healthy patients.
LHON pathology generally occurs in the second or third decade of life and affects predominantly males [4].
However, a relatively rare disorder, Leber's hereditary optic neuropathy (LHON; OMIM 535000), might serve as a promising model system for the experimental analysis of a range of optic nerve diseases.
LHON is a rare genetic disease that without treatment usually leads to blindness.
He tested positive for the 14484T>C mitochondrial mutation associated with LHON.