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Spark Therapeutics files marketing authorization application at EMA for the treatment of Leber’s congenital amaurosis (LCA)
Spark Therapeutics (NASDAQ:ONCE), a gene therapy company based in Philadelphia, USA, has announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency for Investigational “LUXTURNA” (voretigene neparvovec). The gene therapy treatment, designed for patients with vision loss due to Leber’s congenital amurosis, has similarly undergone regulatory submission in the United States. […]Read full story
Phase II results from encapsulated cell therapy technology show benefit in the treatment of patients with macular telangiectasia 2
Results from a Phase II multicentre randomised trial, which had enrolled 67 patients, with macular telangiectasia 2, has reported meeting its primary endpoint, defined as a change in the ellipsoid zone from baseline to month 24, measured by Spectral Domain Optical Coherence Tomography (SD-OCT). According to the sponsor of the study, an increase in the […]Read full story
Reprogramming of Müller glia cells in mammalian retina promotes regeneration of neuronal cells following injury
A US study, published in the journal Nature (Aug 3rd, 2017), has shown that a regenerative gene from zebrafish is capable of driving the generation of neuronal cells in the mammalian retina. Damaged retina in zebrafish results in cells within the eye regenrating new cells to permit the fish to maintain vision, in contrast to […]Read full story
August 4th, 2017: “EURETINA-Brief”© Issue No. 158.
by Dr. Gearóid Tuohy Dear EURETINA Members, A very warm welcome to the August 4th, 2017 edition of EURETINA’s web-based digital magazine, “EURETINA Brief”. EURETINA are delighted to continue our delivery of up-to-date summary briefs on a range of topics of interest to retinal clinicians, specialists and researchers across Europe. This resource is […]Read full story
Whole exome sequencing (WES) continues to yield significant efficiencies in clinical identification of inherited retinal degenerations.
Research results reported by investigators based at the Department of Genetics & Microbiology, University of Barcelona, Spain, have shown that whole exome sequencing (WES) continues to provide efficient returns in the identification of the genetic diagnosis for a range of inherited retinal disorders. The Spanish research group worked on a cohort of 33 families with […]Read full story
Findings from an international genomics consortium almost double the number of associated genetic loci and variants for AMD
Research conducted by the International AMD Genomics Consortium (IAMDGC) has recently reported an almost doubling in the number of genetic risk loci associated with age-related macular degeneration (AMD). In a significant publication in the journal Nature Genetics, the research group has reported on an analysis of >12 million gene variants, “including 163,714 directly genotyped, mostly […]Read full story