Recessive multiple epiphyseal dysplasia and Stargardt disease in two sisters.
Leonardo GatticchiDominika VešelényiováJan MiertusPaolo Enrico MalteseElena ManaraAlisia CostantiniSabrina BenedettiDarina ĎurovčíkováJuraj KrajcovicMatteo BertelliPublished in: Molecular genetics & genomic medicine (2021)
Despite low frequency of occurrence, the detection of multilocus genomic variations in a single disease gene-oriented approach can provide accurate diagnosis even in cases with high phenotypic complexity. A targeted sequencing approach can detect relationships between observed phenotypes and underlying genotypes, useful for clinical management.