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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.
AGN-151587 is an experimental medicine under development for the treatment of Leber congenital amaurosis 10, an inherited form of blindness caused by mutations in the CEP290 gene.
Doctors hope to use the cutting-edge technique to cure the inherited form of blindness known as Leber congenital amaurosis.
(NASDAQ: ISEE) has exercised its option and entered into an exclusive global license agreement with the University of Massachusetts Medical School for rights to develop and commercialize mutation independent novel adeno-associated virus gene therapy product candidates for the treatment of Leber Congenital Amaurosis type 10 (LCA10) due to mutations to the CEP290 gene, the most common type of LCA, the company said.
This American firm founded only in 2013 is the maker of "Luxturna" (Voretigene neparvovec), a gene for treating Leber's congenital amaurosis.
Instead, she and Josh were stunned last month to hear an alarming diagnosis: He has a rare genetic disorder called Leber's hereditary optic neuropathy, also known as Leber's disease.
Among the topics are inheritance patterns, ethical issues, primary congenital glaucoma and juvenile open angle glaucoma, Margan syndrome and other causes of ectopic lentis, familial exudative vitreoretinopathy, retinitis pigmentosa, cone-rod dystrophy, enhanced S-cone syndrome and other NR2ES-related retinal dystrophies, congenital stationary night blindness, optic nerve hypoplasia, and Leber hereditary optic neuropathy.
Leber hereditary optic neuropathy (LHON) is the most common inherited optic neuropathy [1] with the prevalence ranging from 1:31000 to 1:50000 [2].
The session was also attended by Andrew Leber, a PhD student at Harvard University's Department of Government, Alexei Abrahams, research fellow at Princeton University's Niehaus Centre for Globalisation and Governance and Abdulrahman Mohamed al-Shami, associate professor in broadcast journalism in the Department of Mass Communication, Qatar University.