Article
[Gene therapy for hereditary eye diseases].
Ugeskrift for laeger - 7 Nov 2022
Kessel Line, Bertelsen Mette, Klemp Kristian, Christensen Ulrik Correll
Abstract excerpt
Inherited retinal disorders (IRD) are a common cause of severe visual impairment among children and young adults in Denmark. Gene therapy with voretigene neparvovec for a specific, and in Denmark common, cause of IRD (RPE65-related retinal dystrophy) was implemented as standard clinical practice in 2020 as the first of its kind. Twelve Danish patients have been treated with very positive outcomes. Genetically...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
